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Molecular Therapy Methods and Clinical Development Impact Factor: A Comprehensive Guide
Introduction:
The field of molecular therapy is rapidly evolving, offering groundbreaking treatments for previously incurable diseases. Understanding the diverse methods employed and the resulting impact on clinical development is crucial for researchers, clinicians, and investors alike. This comprehensive guide delves into the core molecular therapy techniques, analyzes their influence on clinical trial success rates, and explores the key metrics used to assess their impact. We'll dissect the concept of "impact factor" in the context of molecular therapy publications and examine how advancements in this field are shaping the future of medicine. Prepare to gain a deeper understanding of this transformative area of medical research.
1. Exploring Key Molecular Therapy Methods:
Molecular therapies represent a paradigm shift in medicine, moving beyond traditional treatments to target the root cause of diseases at a molecular level. Several key methods are driving this revolution:
Gene Therapy: This involves introducing genetic material into cells to treat or prevent disease. This can include gene replacement (correcting faulty genes), gene silencing (inactivating harmful genes), or gene editing (precisely altering DNA sequences). Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, are commonly used to deliver therapeutic genes. Non-viral methods, including liposomes and nanoparticles, are also under development.
Cell Therapy: This involves using cells to treat diseases. This includes the use of stem cells (capable of differentiating into various cell types) to repair damaged tissues, adoptive cell therapies (like CAR T-cell therapy) that harness the body's immune system to fight cancer, and cell-based drug delivery systems.
RNA Interference (RNAi): RNAi utilizes small RNA molecules (siRNA or miRNA) to silence specific genes by degrading their messenger RNA (mRNA) or inhibiting translation. This approach holds promise for treating a wide range of diseases, including cancer and genetic disorders.
Antisense Oligonucleotides (ASOs): ASOs are short, single-stranded DNA or RNA sequences designed to bind to specific mRNA molecules, preventing protein synthesis. They offer a targeted approach to modulate gene expression and have shown efficacy in treating various disorders.
2. Clinical Development: From Bench to Bedside:
Translating molecular therapies from the laboratory to clinical practice requires a rigorous process involving multiple phases of clinical trials. These phases progressively evaluate the safety and efficacy of the therapy in larger and more diverse populations:
Preclinical Studies: Before human trials, extensive preclinical research is conducted in vitro (in cell cultures) and in vivo (in animal models) to assess the therapy's safety, efficacy, and mechanism of action.
Phase I Trials: These initial trials focus primarily on safety and determining the optimal dosage. A small number of healthy volunteers or patients are involved.
Phase II Trials: Phase II trials evaluate the efficacy of the therapy in a larger group of patients with the target disease. Researchers also continue to monitor safety and side effects.
Phase III Trials: These large-scale trials compare the new molecular therapy to existing treatments or a placebo. The results determine whether the therapy is effective and safe enough for widespread use.
Post-Market Surveillance: Even after approval, ongoing monitoring is crucial to detect any unexpected long-term effects.
3. Impact Factor and its Relevance in Molecular Therapy:
The impact factor of a journal is a metric reflecting the average number of citations received by articles published in that journal over a specific period. A high impact factor generally suggests that the journal publishes highly influential research. In the field of molecular therapy, journals with high impact factors, such as Molecular Therapy, Nature Biotechnology, and The New England Journal of Medicine, play a crucial role in disseminating groundbreaking research findings and shaping the direction of clinical development. The impact factor of a publication can indirectly reflect the importance and influence of the research presented, influencing funding opportunities and shaping clinical practice. However, it's crucial to remember that impact factor is just one metric and shouldn't be the sole determinant of research quality.
4. Challenges and Future Directions:
Despite the significant progress, several challenges remain in molecular therapy:
Delivery Challenges: Efficient and targeted delivery of therapeutic molecules to the desired cells or tissues remains a major hurdle.
Immunogenicity: Some therapeutic molecules, particularly viral vectors, can trigger an immune response, limiting their efficacy and safety.
Cost and Accessibility: Many molecular therapies are expensive, making them inaccessible to many patients.
Long-term effects: The long-term effects of some molecular therapies are still unknown, necessitating ongoing monitoring and research.
Future research will likely focus on developing safer and more effective delivery systems, improving targeting strategies, and reducing costs to ensure wider accessibility. Advances in gene editing technologies, such as CRISPR-Cas9, hold immense promise for treating a wider range of diseases.
5. Case Studies Highlighting Impact:
Several successful clinical trials showcasing the impact of molecular therapies on clinical development include:
CAR T-cell therapy for leukemia: This revolutionary approach has shown remarkable success in treating certain types of leukemia, demonstrating the potential of cell-based therapies.
Spinraza for spinal muscular atrophy: This ASO therapy has significantly improved the lives of patients with this devastating genetic disorder, highlighting the power of targeted gene silencing.
Luxturna for inherited retinal dystrophy: This gene therapy has restored vision in some patients with this blinding condition, showcasing the transformative potential of gene replacement therapy. These successes underscore the growing impact of molecular therapies and encourage further research and development.
Article Outline: Molecular Therapy Methods and Clinical Development Impact Factor
Name: A Deep Dive into Molecular Medicine: Methods, Clinical Translation, and Impact
Introduction: Overview of molecular therapy and the significance of its impact factor.
Chapter 1: Detailed explanation of various molecular therapy methods (gene therapy, cell therapy, RNAi, ASOs).
Chapter 2: Comprehensive overview of clinical development stages and regulatory pathways.
Chapter 3: In-depth analysis of the impact factor concept and its relevance in evaluating molecular therapy research.
Chapter 4: Discussion of current challenges and future directions in the field.
Chapter 5: Case studies highlighting the successful clinical translation of molecular therapies.
Conclusion: Summary of key takeaways and future prospects of molecular therapies.
(The detailed content for each chapter is provided above in the main article.)
FAQs:
1. What is the difference between gene therapy and cell therapy? Gene therapy modifies genes within cells, while cell therapy uses cells as the therapeutic agent.
2. How are viral vectors used in gene therapy? Viral vectors deliver therapeutic genes into target cells.
3. What are the phases of clinical trials for molecular therapies? Preclinical studies, Phase I, Phase II, Phase III, and post-market surveillance.
4. What is the impact factor of a journal, and why is it important? It reflects the average number of citations received by articles in that journal, indicating its influence.
5. What are the challenges in developing molecular therapies? Delivery, immunogenicity, cost, and long-term effects.
6. What are some examples of successful molecular therapies? CAR T-cell therapy, Spinraza, and Luxturna.
7. How is CRISPR-Cas9 technology impacting molecular therapy? It offers precise gene editing capabilities.
8. What role do regulatory agencies play in clinical development? They ensure the safety and efficacy of therapies before approval.
9. Where can I find more information on molecular therapy research? Reputable journals (e.g., Molecular Therapy, Nature Biotechnology) and databases (e.g., PubMed).
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1. Advances in Gene Editing Technologies for Molecular Therapies: Discusses the latest developments in CRISPR and other gene editing tools.
2. Viral Vector Engineering for Enhanced Gene Therapy Delivery: Focuses on improving the efficiency and safety of viral vectors.
3. The Role of Nanotechnology in Molecular Therapy Drug Delivery: Explores the use of nanomaterials for targeted drug delivery.
4. CAR T-Cell Therapy: Clinical Applications and Future Directions: Details the applications and challenges of this innovative cancer therapy.
5. RNA Interference: Mechanisms, Applications, and Challenges: Provides a comprehensive overview of RNAi technology.
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7. The Economics of Molecular Therapies: Challenges and Opportunities: Discusses the cost-effectiveness and accessibility of these treatments.
8. Ethical Considerations in Molecular Therapy Research and Development: Explores the ethical implications of these powerful technologies.
9. Patient Advocacy and Access to Innovative Molecular Therapies: Highlights the importance of patient advocacy in ensuring access to new treatments.
| molecular therapy methods and clinical development impact factor: Cell and Gene Therapies Miguel-Angel Perales, Syed A. Abutalib, Catherine Bollard, 2018-11-27 In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee. |
| molecular therapy methods and clinical development impact factor: From Research to Reality The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada, 2020-11-05 From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them. |
| molecular therapy methods and clinical development impact factor: Stem Cells Mariusz Z. Ratajczak, 2020-01-02 Since different types of stem cells for therapeutic applications have recently been proposed, this timely volume explores various sources of stem cells for tissue and organ regeneration and discusses their advantages and limitations. Also discussed are pros and cons for using embryonic stem cells, induced pluripotent stem cells, and adult stem cells isolated from postnatal tissues. Different types of adult stem cells for therapeutic applications are also reviewed, including hematopoietic stem cells, epidermal stem cells, endothelial progenitors, neural stem cells, mesenchymal stem cells, and very small embryonic-like stem cells. This book also addresses paracrine effects of stem cells in regenerative medicine that are mediated by extracellular microvesicles and soluble secretome. Finally, potential applications of stem cells in cardiology, gastroenterology, neurology, immunotherapy, and aging are presented. This is an ideal book for students and researchers working in the stem cell research field. |
| molecular therapy methods and clinical development impact factor: Stem Cells Christine L. Mummery, Anja van de Stolpe, Bernard Roelen, Hans Clevers, 2014-05-23 The second edition of Stem Cells: Scientific Facts and Fiction provides the non-stem cell expert with an understandable review of the history, current state of affairs, and facts and fiction of the promises of stem cells. Building on success of its award-winning preceding edition, the second edition features new chapters on embryonic and iPS cells and stem cells in veterinary science and medicine. It contains major revisions on cancer stem cells to include new culture models, additional interviews with leaders in progenitor cells, engineered eye tissue, and xeno organs from stem cells, as well as new information on organs on chips and adult progenitor cells. In the past decades our understanding of stem cell biology has increased tremendously. Many types of stem cells have been discovered in tissues that everyone presumed were unable to regenerate in adults, the heart and the brain in particular. There is vast interest in stem cells from biologists and clinicians who see the potential for regenerative medicine and future treatments for chronic diseases like Parkinson's, diabetes, and spinal cord lesions, based on the use of stem cells; and from entrepreneurs in biotechnology who expect new commercial applications ranging from drug discovery to transplantation therapies. - Explains in straightforward, non-specialist language the basic biology of stem cells and their applications in modern medicine and future therapy - Includes extensive coverage of adult and embryonic stem cells both historically and in contemporary practice - Richly illustrated to assist in understanding how research is done and the current hurdles to clinical practice |
| molecular therapy methods and clinical development impact factor: Development of Gene Therapies Avery McIntosh, Oleksandr Sverdlov, 2024-05-23 Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx Insights into commercial models, access hurdles, and health economics of gene therapies Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective |
| molecular therapy methods and clinical development impact factor: The Psychology of Fake News Rainer Greifeneder, Mariela Jaffe, Eryn Newman, Norbert Schwarz, 2020-08-13 This volume examines the phenomenon of fake news by bringing together leading experts from different fields within psychology and related areas, and explores what has become a prominent feature of public discourse since the first Brexit referendum and the 2016 US election campaign. Dealing with misinformation is important in many areas of daily life, including politics, the marketplace, health communication, journalism, education, and science. In a general climate where facts and misinformation blur, and are intentionally blurred, this book asks what determines whether people accept and share (mis)information, and what can be done to counter misinformation? All three of these aspects need to be understood in the context of online social networks, which have fundamentally changed the way information is produced, consumed, and transmitted. The contributions within this volume summarize the most up-to-date empirical findings, theories, and applications and discuss cutting-edge ideas and future directions of interventions to counter fake news. Also providing guidance on how to handle misinformation in an age of “alternative facts”, this is a fascinating and vital reading for students and academics in psychology, communication, and political science and for professionals including policy makers and journalists. |
| molecular therapy methods and clinical development impact factor: Sharing Clinical Trial Data Institute of Medicine, Board on Health Sciences Policy, Committee on Strategies for Responsible Sharing of Clinical Trial Data, 2015-04-20 Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research-from funders, to researchers, to journals, to physicians, and ultimately, to patients. |
| molecular therapy methods and clinical development impact factor: Introduction to Digital Audio Coding and Standards Marina Bosi, Richard E. Goldberg, 2012-12-06 Introduction to Digital Audio Coding and Standards provides a detailed introduction to the methods, implementations, and official standards of state-of-the-art audio coding technology. In the book, the theory and implementation of each of the basic coder building blocks is addressed. The building blocks are then fit together into a full coder and the reader is shown how to judge the performance of such a coder. Finally, the authors discuss the features, choices, and performance of the main state-of-the-art coders defined in the ISO/IEC MPEG and HDTV standards and in commercial use today. The ultimate goal of this book is to present the reader with a solid enough understanding of the major issues in the theory and implementation of perceptual audio coders that they are able to build their own simple audio codec. There is no other source available where a non-professional has access to the true secrets of audio coding. |
| molecular therapy methods and clinical development impact factor: Hot Topics in Cell Biology José Becerra, Leonor Santos-Ruiz, 2012 Summary This book is a definitive overview of the current 'state of the art' in cell biology. It is based on papers presented by leading researchers at the Spanish Society for Cell Biology's XIV Congress - a Congress that strives to achieve scientific excellence. Each participant was asked to prepare a 'mini review' of current and likely future development in their area of research. This book is based on those reviews. As such, it is therefore an analysis of current and future trends. Key Features Contains contributions from some of the world's leading researchers. The book is multidisciplinary, covering almost all topics in cell biology: from basic to applied cell biology, and a wide variety of models: from in vitro to vivo models, ranging from fish to rodents and humans. Each 'mini review' is an easy-read piece, describing the state of the art on a topic with clear language and in a summary format. The mini review format makes the book attractive not only to readers involved in cell biology research and teaching, but also professionals from other disciplines and students. The book takes a truly multidisciplinary approach; it covers a wide array of topics, and the book reflects how cell biology interacts with other disciplines The Editors Jose Becerra is Professor of Cell Biology at the University of Málaga (Spain) since 1989. He has been Dean Secretary, Vice-Dean and Dean of the Faculty of Sciences of Málaga, and is now the Head of the Department of Cell Biology, Genetics and Physiology. From 2001 to 2003 he was the Director of the Andalusian Laboratory of Biology (LAB, Seville), which was converted in the Andalusian Centre for Developmental Biology (CABD) under his term. He is a member of the Technical Committee of the National Stem Cell Bank since 2007, patron of the Board of Trustees of IMABIS Foundation (Mediterranean Institute for the Advance of Biotechnology and Health Research), coordinator of the Biomaterials and Tissue Engineering Area of the the Biomedical Research Networking Center in Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), and member of the Direction Committee of the CIBER-BBN. Leonor Santos-Ruiz is Senior Researcher of the CIBER-BBN network at the Andalusian Center for Nanomedicine and Biotechnology (BIONAND). She started her career studying the cellular and molecular basis of lower vertebrates' amazing ability for tissue regeneration, with a special attention to bone and spinal cord repair. Readership Cell biology academics and researchers Contents Introduction Dynamics of cell compartments The intracellular trafficking Cell signaling Autophagy, apoptosis and cell homeostasis Cell biology of aging Plant cell biology Methods in cell biology Applied cell biology Cell biology of cancer Cell therapies and tissue engineering Neurodegeneration and cell biology Nanotechnology and cell biology: challenges and opportunities |
| molecular therapy methods and clinical development impact factor: Improving Healthcare Quality in Europe Characteristics, Effectiveness and Implementation of Different Strategies OECD, World Health Organization, 2019-10-17 This volume, developed by the Observatory together with OECD, provides an overall conceptual framework for understanding and applying strategies aimed at improving quality of care. Crucially, it summarizes available evidence on different quality strategies and provides recommendations for their implementation. This book is intended to help policy-makers to understand concepts of quality and to support them to evaluate single strategies and combinations of strategies. |
| molecular therapy methods and clinical development impact factor: The Centrosome in Cell Replication and Early Development , 2000-09-26 Centrosomes play an integral role in the growth of cells and the ultimate development of many animals, and sometimes plants. In addition to the normal growth of cells, centrosomes can also play a key role in the spread of cancer and are of increased interest to both the genetics and oncology communities. Volume 49 of Current Topics in Developmental Biology will present all known research surrounding the centrosome, across a variety of systems, will be well referenced, and speculate where the research is headed. - Discusses centrosomes and cancer, centrosomes and early development, and molecular biology of the centrosome - Heavily illustrated, with many color figures - Chapters written by international leaders in the field |
| molecular therapy methods and clinical development impact factor: Modern Methods of Clinical Investigation Institute of Medicine, Committee on Technological Innovation in Medicine, 1990-02-01 The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians. |
| molecular therapy methods and clinical development impact factor: New Trends in Biotechnology Nanjappa Shamanna Subba Rao, 1992 |
| molecular therapy methods and clinical development impact factor: Novel Gene Therapy Approaches Ming Wei, David Good, 2013-02-13 Gene therapy has the potential to be a tailor-made therapeutic with increased specificity and decreased side effects that can offer a cure for many disorders. The aim of this book is to provide up-to-date reviews of the rapidly growing field of gene therapy. Chapters cover a large range of topics including methods of gene delivery, and identification of targets with several papers on cancer gene therapy. If more people become aware of the true nature and potential of gene therapy, perhaps we can achieve the full benefit of such an innovative approach for the treatment of a range of diseases, including cancer. |
| molecular therapy methods and clinical development impact factor: Improving and Accelerating Therapeutic Development for Nervous System Disorders Institute of Medicine, Board on Health Sciences Policy, Forum on Neuroscience and Nervous System Disorders, 2014-02-06 Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials. |
| molecular therapy methods and clinical development impact factor: Current Protocols in Molecular Biology , |
| molecular therapy methods and clinical development impact factor: The Role of NIH in Drug Development Innovation and Its Impact on Patient Access National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Board on Health Care Services, 2020-01-27 To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24â€25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop. |
| molecular therapy methods and clinical development impact factor: ECODESIGN Pilot Wolfgang Wimmer, Rainer Züst, 2003-01-31 Up to now, environmental policies have relied mainly on rules, regulations, and prohibitions. This kind of environmental policy -- a clearly reactive approach – has shown a mounting array of limitations. There is a clear need to seek new solutions, in particular those involving the initiative of firms themselves. Since the mid-1980’s, a new approach called “environmental management” has been established in research and practical applications; it is designed to systematically integrate environmental considerations into company activities. If, for instance, environmental objectives are being formulated for an enterprise, it is imperative to identify, highlight, and analyze real and significant environmental impacts of the firm's activities and to take appropriate measures to improve its performance. Controlling and implementing these environmental objectives requires suitable structures, procedures, and tools. Many motivated companies have approached the issue of environmental management through incorporating methods such “continual improvement processes” and “Life Cycle Assessment” into the various operational activities of the enterprise. Some firms initiated such transformation processes at their own production sites because it was easier to identify their own environmental impacts than to analyze, let alone mitigate, the effects of upstream and downstream processes. However, in many cases these processes are decisive factors in the overall environmental performance of a product. It has become clear that product design can influence these processes to a great extent. |
| molecular therapy methods and clinical development impact factor: Oversight and Review of Clinical Gene Transfer Protocols Institute of Medicine, Board on Health Sciences Policy, Committee on the Independent Review and Assessment of the Activities of the NIH Recombinant DNA Advisory Committee, 2014-03-27 Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes. |
| molecular therapy methods and clinical development impact factor: Handbook of Research on Advancements in Cancer Therapeutics Kumar, Sumit, Rizvi, Moshahid Alam, Verma, Saurabh, 2020-11-27 The complexity of cancer demands an integrated approach from both a cancer biology standpoint and a pharmaceutical basis to understand the different anticancer modalities. Current research has been focused on conventional and newer anticancer modalities, recent discoveries in cancer research, and also the advancements in cancer treatment. There is a current need for more research on the advances in cancer therapeutics that bridge the gap between basic research (pharmaceutical drug development processes, regulatory issues, and translational experimentation) and clinical application. Recent promising discoveries such as immunotherapies, promising therapies undergoing clinical trials, synthetic lethality, carbon beam radiation, and other exciting targeted therapies are being studied to improve and advance the studies of modern cancer treatment. The Handbook of Research on Advancements in Cancer Therapeutics serves as a comprehensive guide in modern cancer treatment by combining and merging the knowledge from both cancer biology and the pharmacology of anticancer modalities. The chapters come from multi-disciplinary backgrounds, including scientists and clinicians from both academia and various industries, to discuss nascent personalized therapies and big data-driven cancer treatment. While highlighting topic areas that include cancer prevention, cancer therapeutics, and cancer treatments through the lenses of technology, medicine/drugs, and alternate therapies, this book is ideally intended for oncologists, radiation oncologists, surgical oncologists, and cancer biologists, along with practitioners, stakeholders, researchers, academicians, and students who are interested in understanding the most fundamental aspects of cancer and the available therapeutic opportunities. |
| molecular therapy methods and clinical development impact factor: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne |
| molecular therapy methods and clinical development impact factor: Network Pharmacology Shao Li, 2021-09-29 This book introduces “network pharmacology” as an emerging frontier subject of systematic drug research in the era of artificial intelligence and big data. Network Pharmacology is an original subject of fusion system biology, bioinformatics, network science and other related disciplines. It emphasizes on starting from the overall perspective of the system level and biological networks, the analysis of the laws of molecular association between drugs and their treatment objects, reveals the systematic pharmacological mechanisms of drugs, and guides the research and development of new drugs and clinical diagnosis and treatment. After it was proposed, network pharmacology has been paid attention by researchers, and it has been rapidly developed and widely used. In order to systematically reveal the biological basis of diagnosis and treatment in traditional Chinese medicine and modern medicine, we proposed a new concept of network target for the first time, which has become the core theory of network pharmacology. The core principle of a network target is to construct a biological network that can be used to decipher complex diseases. The network is then used as the therapeutic target, to which multicomponent remedies are applied. This book mainly includes four parts: 1) The concept and theory of network pharmacology; 2) Common analysis methods, databases and software in network pharmacological research; 3) Typical cases of traditional Chinese medicine modernization and modern drug research based on network pharmacology; 4) Network pharmacology practice process based on drugs and diseases. |
| molecular therapy methods and clinical development impact factor: Molecular and Cellular Therapeutics David Whitehouse, Ralph Rapley, 2012-02-17 Molecular and Cellular Therapeutics aims to bring together key developments in the areas of molecular diagnostics, therapeutics and drug discovery. The book covers topics including diagnostics, therapeutics, model systems, clinical trials and drug discovery. The developing approaches to molecular and cellular therapies, diagnostics and drug discovery are presented in the context of the pathologies they are devised to treat. |
| molecular therapy methods and clinical development impact factor: In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders Houria Bachtarzi, 2019-03-13 Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis. |
| molecular therapy methods and clinical development impact factor: EU Lobbying: Empirical and Theoretical Studies David Coen, 2013-10-31 EU Lobbying: Empirical and Theoretical studies offers an analysis of large empirical studies of interest group politics and Lobbying in Europe. Recognising the continued European economic integration, globalisation and the changing role of the state, it observs significant adaptations in interest mobilisation and strategic behavour. This book assesses the logic of collective and direct action, the logic of access and influence, the logic of venue-shopping and alliance building. It addresses specific issues such as: the emergence of elite pluralism in EU institutions, the pump priming of political action by EU institutions, and the growing political sophistication of private and public interests in Brussels. Through these issues the book explores how interest groups lobby different European institutions along the policy process and how the nature of policy dictates the style and level of lobbying. This book was previously published as a special issue of Jounal of European Public Policy |
| molecular therapy methods and clinical development impact factor: Toxicological Profile for Lead , 2007 |
| molecular therapy methods and clinical development impact factor: Cellular and Molecular Immunology Abul K. Abbas, Andrew H. Lichtman, 2005 The 5th Edition of this comprehensive title continues the tradition of delivering an accessible, engaging, and current introduction to this essential subject. The authors describe the principles of basic and applied immunology in a concise, straightforward manner, while incorporating the most up-to-date information. Over 400 illustrations help readers quickly and easily grasp key concepts. The entire text has been revised and includes new information about the organization of lymphoid organs and the mechanisms of innate immunity. (Midwest). |
| molecular therapy methods and clinical development impact factor: Muscle Gene Therapy Dongsheng Duan, 2009-11-26 Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy. |
| molecular therapy methods and clinical development impact factor: Phenotypic Plasticity & Evolution David W. Pfennig, 2021-05-31 Phenotypic plasticity – the ability of an individual organism to alter its features in direct response to a change in its environment – is ubiquitous. Understanding how and why this phenomenon exists is crucial because it unites all levels of biological inquiry. This book brings together researchers who approach plasticity from diverse perspectives to explore new ideas and recent findings about the causes and consequences of plasticity. Contributors also discuss such controversial topics as how plasticity shapes ecological and evolutionary processes; whether specific plastic responses can be passed to offspring; and whether plasticity has left an important imprint on the history of life. Importantly, each chapter highlights key questions for future research. Drawing on numerous studies of plasticity in natural populations of plants and animals, this book aims to foster greater appreciation for this important, but frequently misunderstood phenomenon. Key Features Written in an accessible style with numerous illustrations, including many in color Reviews the history of the study of plasticity, including Darwin’s views Most chapters conclude with recommendations for future research |
| molecular therapy methods and clinical development impact factor: Current Trends in Cancer Research , 1964 |
| molecular therapy methods and clinical development impact factor: Cardiac Gene Therapy Kiyotake Ishikawa, 2016-12-02 This detailed book provides methodological information on cardiac gene delivery, from classic to state-of-the-art technologies and techniques. Efficient, cardiac-specific, and safe vectors, as well as refined vector delivery methods, are key for successful cardiac gene transfer and eventually for improving patients’ outcomes. Newer vectors and more efficient vector delivery methods have the potential to dramatically improve gene transduction efficacy, while novel gene manipulation techniques enforce the therapeutic power and broaden disease targets. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Cardiac Gene Therapy: Methods and Protocols serves as a valuable tool for molecular biologists and physiologists in the cardiology field conducting cardiac gene transfer research, which will ultimately lead to further advancements in the vital field. |
| molecular therapy methods and clinical development impact factor: Pain Management and the Opioid Epidemic National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Committee on Pain Management and Regulatory Strategies to Address Prescription Opioid Abuse, 2017-09-28 Drug overdose, driven largely by overdose related to the use of opioids, is now the leading cause of unintentional injury death in the United States. The ongoing opioid crisis lies at the intersection of two public health challenges: reducing the burden of suffering from pain and containing the rising toll of the harms that can arise from the use of opioid medications. Chronic pain and opioid use disorder both represent complex human conditions affecting millions of Americans and causing untold disability and loss of function. In the context of the growing opioid problem, the U.S. Food and Drug Administration (FDA) launched an Opioids Action Plan in early 2016. As part of this plan, the FDA asked the National Academies of Sciences, Engineering, and Medicine to convene a committee to update the state of the science on pain research, care, and education and to identify actions the FDA and others can take to respond to the opioid epidemic, with a particular focus on informing FDA's development of a formal method for incorporating individual and societal considerations into its risk-benefit framework for opioid approval and monitoring. |
| molecular therapy methods and clinical development impact factor: A Therapy Technique for Improving Memory Jennifer A. Brush, 1998-01-01 |
| molecular therapy methods and clinical development impact factor: Germline Stem Cells Steven X. Hou, Shree Ram Singh, 2014-10-15 In this comprehensive and cutting-edge book, leading experts explore the parameters that define germline stem cells and the mechanisms that regulate the cell behavior in order to better isolate, characterize and maintain them. The volume begins by providing protocols for germline stem cell identification and regulation in model organisms, and concludes with detailed chapters covering current techniques involving in vitro culture and the applications of the cells. |
| molecular therapy methods and clinical development impact factor: Clinical Pharmacy and Therapeutics Roger Walker (Ph. D.), Clive Edwards, 2003 The new edition of this popular, well-established textbook addresses the expanding role of the pharmacist in treating patients. It covers treatment of common diseases as well as other medical, therapeutic and patient related issues. Written by both pharmacists and clinicians to reflect a team approach, it offers an in-depth analysis of drug therapy in the treatment of disease, relying on input from the pharmacist as a member of the team in hospital and community settings. Information is easy to locate in a logical format organized primarily by systems and disorders. |
| molecular therapy methods and clinical development impact factor: Molecular Diagnostics Lela Buckingham, 2012 The first text on molecular diagnostics specifically designed for clinical laboratory science programs is back! This exceptional resource introduces the fundamentals of nucleic acid, as well as more advanced concepts. With a focus on the application of molecular concepts in the clinical laboratory to diagnosis diseases, the 2nd Edition includes important updates and improvements to keep up with the rapidly developing field. Inside youll find in-depth explanations of the principles of molecular-based assays as well as reference material, trouble-shooting tips for the laboratory, and discussions that emphasize the continuing emergence of new diagnostic technologies. |
| molecular therapy methods and clinical development impact factor: Molecular Analyses Scott Orland Rogers, 2022-07-11 DNA and RNA extraction methods from a variety of tissues and samples are now routine, including extraction from single cells. Many methods are now automated. Sequencing efficiency has reached the point where it is now possible to obtain gigabases of data, both quickly and inexpensively. Such methods permit the identification of gene versions, including those associated with disease (e.g. small nucleotide polymorphism analyses, or SNPs). The general public as well as clinicians can now access a wide variety of literature on the molecular bases of diseases, allowing them to better assess disease risks and treatments. This volume concentrates on medically-focused methods, and therefore the major audience will be medical professionals, students, and those involved in medically-related research endeavors. There are also papers in this volume dealing specifically with methods developed to analyze large sequence data sets. Many methods reviewed herein are more broadly applicable to other fields in biology, chemistry, bioinformatics, and bioengineering, and are intended for a broad readership. Key Features Summarizes nucleic acid extractions from a wide variety of tissues and cells Describes processes of nucleic acid preservation Reviews forensic sampling, detection of nucleic acids, and delivery of nucleic acids to multicellular organisms Provides essential guidance for sequencing, sequence analysis, database searches, and phylogenetic analyses Includes additional methods useful for analysis of nucleic acids and proteins Related Titles DeSalle, et al. Phylogenomics: A Primer (ISBN 978-0-3670-2849-7). Jennings, W. B. Phylogenomic Data Acquisition: Principles and Practice (ISBN 978-0-3678-6980-9). Wang, X. Next-Generation Sequencing Data Analysis (ISBN 978-1-4822-1788-9) Sung, W.-K. Algorithms for Next-Generation Sequencing (ISBN 978-0-3676-5797-0) |
| molecular therapy methods and clinical development impact factor: Environmental Epigenetics L. Joseph Su, Tung-chin Chiang, 2015-05-18 This book examines the toxicological and health implications of environmental epigenetics and provides knowledge through an interdisciplinary approach. Included in this volume are chapters outlining various environmental risk factors such as phthalates and dietary components, life states such as pregnancy and ageing, hormonal and metabolic considerations and specific disease risks such as cancer cardiovascular diseases and other non-communicable diseases. Environmental Epigenetics imparts integrative knowledge of the science of epigenetics and the issues raised in environmental epidemiology. This book is intended to serve both as a reference compendium on environmental epigenetics for scientists in academia, industry and laboratories and as a textbook for graduate level environmental health courses. Environmental Epigenetics imparts integrative knowledge of the science of epigenetics and the issues raised in environmental epidemiology. This book is intended to serve both as a reference compendium on environmental epigenetics for scientists in academia, industry and laboratories and as a textbook for graduate level environmental health courses. |
| molecular therapy methods and clinical development impact factor: Regenerative Medicine Tingting Qiu, Mondher Toumi, 2023-05-23 A comprehensive review of the challenges that exist in patient accessibility to regenerative medicines (RMs), presenting clinical trials, marketing authorization, HTA, pricing, reimbursement, affordability, payment and partnership agreements of RMs and commercialization. Specfically, we investigated how COVID-19 has impacted the RM industry by elaborating on the disruptions it caused but also the new opportunities it brought. The ultimate goal of this work is to make strategic recommendations for manufacturers and decisions-makers on effective strategies to address the above obstacles and facilitate patient access to promising regenerative medicines. FEATURES Regenerative medicine (RM) is an emerging interdisciplinary field aiming to replace or regenerate human cells, tissues, or organs in order to restore normal function. RM holds the promise of revolutionizing treatment in the 21st century. RMs bring new hope for some previously untreatable diseases, as well as holding promise for the treatment of common chronic diseases. Rapid advancements in biotechnology and improved understanding of disease pathophysiology have attracted tremendous interests in the development of RMs. Discusses the high cost of RMs which may challenge the sustainability of healthcare insurers (public and private). |
| molecular therapy methods and clinical development impact factor: Regenerative Pharmacology George J. Christ, Karl-Erik Andersson, 2013-04-15 A state-of-the-art primer on the role of pharmacological sciences in regenerative medicine, for advanced students, postdoctoral fellows, and researchers. |
