Application of Innovative Techniques in Genetic and Cellular Therapies

Application of Innovative Techniques in Genetic and Cellular Therapies PDF Author: Yuan Xiong
Publisher: Frontiers Media SA
ISBN: 2832536506
Category : Technology & Engineering
Languages : en
Pages : 136

Get Book Here

Book Description
Tightly linked to tissue engineering and regenerative medicine, genetic and cellular therapies have drawn universal attention. Since genetic sequencing is critical for precision medicine, the combination of bioinformatics and therapies is important to conduct prescriptive and predictive analytics based on genetic sequencing data. Bioinformatics and computational approaches have been widely considered for the development of genetic and cellular therapies. Therefore, the current research topic aims to cover recent advances in studies such as functional genomics, proteomics, metabolomics, and bioinformatics that bring in new perspectives on genetic therapies. The central goal of regenerative medicine is to replace damaged or diseased tissue with cells that are integrated and functioning optimally. Stem cell-based therapy is restricted by the limited availability of cell sources, the excessive cost, and the anticipated difficulties of clinical translation and ethical approval. Some promising alternatives are to incite the patient's innate ability of tissue repairs such as in situ tissue regeneration, which has the potential to provide new therapeutic options for tissue engineering and regenerative medicine. These innovative cellular therapies are also contained in our research topic.

Application of Innovative Techniques in Genetic and Cellular Therapies

Application of Innovative Techniques in Genetic and Cellular Therapies PDF Author: Yuan Xiong
Publisher: Frontiers Media SA
ISBN: 2832536506
Category : Technology & Engineering
Languages : en
Pages : 136

Get Book Here

Book Description
Tightly linked to tissue engineering and regenerative medicine, genetic and cellular therapies have drawn universal attention. Since genetic sequencing is critical for precision medicine, the combination of bioinformatics and therapies is important to conduct prescriptive and predictive analytics based on genetic sequencing data. Bioinformatics and computational approaches have been widely considered for the development of genetic and cellular therapies. Therefore, the current research topic aims to cover recent advances in studies such as functional genomics, proteomics, metabolomics, and bioinformatics that bring in new perspectives on genetic therapies. The central goal of regenerative medicine is to replace damaged or diseased tissue with cells that are integrated and functioning optimally. Stem cell-based therapy is restricted by the limited availability of cell sources, the excessive cost, and the anticipated difficulties of clinical translation and ethical approval. Some promising alternatives are to incite the patient's innate ability of tissue repairs such as in situ tissue regeneration, which has the potential to provide new therapeutic options for tissue engineering and regenerative medicine. These innovative cellular therapies are also contained in our research topic.

Gene and Cell Therapy

Gene and Cell Therapy PDF Author: Nancy Smyth Templeton
Publisher: CRC Press
ISBN: 084938799X
Category : Science
Languages : en
Pages : 1154

Get Book Here

Book Description
Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Primary and Stem Cells

Primary and Stem Cells PDF Author: Uma Lakshmipathy
Publisher: John Wiley & Sons
ISBN: 0470610743
Category : Science
Languages : en
Pages : 314

Get Book Here

Book Description
This book describes basic cell engineering methods, emphasizing stem cell applications, and use of the genetically modified stem cells in cell therapy and drug discovery. Together, the chapters introduce and offer insights on new techniques for engineering of stem cells and the delivery of transgenes into stem cells via various viral and non-viral systems. The book offers a guide to the types of manipulations currently available to create genetically engineered stem cells that suit any investigator's purpose, whether it's basic science investigation, creation of disease models and screens, or cells for therapeutic applications.

Gene and Cell Therapy: Biology and Applications

Gene and Cell Therapy: Biology and Applications PDF Author: Giridhara R. Jayandharan
Publisher: Springer
ISBN: 9811304815
Category : Medical
Languages : en
Pages : 316

Get Book Here

Book Description
Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics.

Second Generation Cell and Gene-Based Therapies

Second Generation Cell and Gene-Based Therapies PDF Author: Alain Vertes
Publisher: Academic Press
ISBN: 0128120339
Category : Science
Languages : en
Pages : 846

Get Book Here

Book Description
Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products. The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization "Perspectives" section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies

Gene and Cell Therapy

Gene and Cell Therapy PDF Author: Giridhara R. Jayandharan
Publisher:
ISBN: 9789811304828
Category : HEALTH & FITNESS
Languages : en
Pages : 305

Get Book Here

Book Description
Recent advances in stem cell biology, nanotechnology and gene therapy have opened new avenues for therapeutics. The availability of molecular therapeutics that rely on the delivery of DNA, RNA or proteins, harnessing enhanced delivery with nanoparticles, and the regenerative potential of stem cells (adult, embryonic or induced pluripotent stem cells) has had a tremendous impact on translational medicine. The chapters in this book cover a range of strategies for molecular and cellular therapies for human disease, their advantages, and central challenges to their widespread application. Potential solutions to these issues are also discussed in detail. Further, the book addresses numerous advances in the field of molecular therapeutics that will be of interest to the general scientific community. Lastly, the book provides specific examples of disease conditions for which these strategies have been transferred to the clinic. As such, it will be extremely useful for all students, researchers and clinicians working in the field of translational medicine and molecular therapeutics.

Manufacturing of Gene Therapeutics

Manufacturing of Gene Therapeutics PDF Author: G. Subramanian
Publisher: Springer Science & Business Media
ISBN: 1461513537
Category : Medical
Languages : en
Pages : 355

Get Book Here

Book Description
Advances in molecular biology and recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches and methods are readily available for gene cloning and therapeutic vector construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus gene therapy has already shown that it holds great promise for the treatment of many diseases and disorders. In general it involves the delivery of recombinant genes or transgenes into somatic cells to replace proteins with a genetic defect or to transfer with the pathological process of an illness. The viral and non-viral delivery systems may hold the potential for future non-invasive, cost-effective oral therapy of genetically-based disorders. Recent years have seen considerable progress in the discovery and early clinical development of a variety of gene therapeutic products. The availability, validation, and implementation of gene therapeutic products has also enabled success in testing and evaluation. New challenges will need to be overcome to ensure that products will also be successful in later clinical development and ultimately for marketing authorisation. These new challenges will include improvements in delivery systems, better control of in-vivo targeting, increased level transduction and duration of expression of the gene, and manufacturing process efficiencies that enable reduction in production costs. Perhaps profound understanding of regulated gene design may result in innovative bioproducts exhibiting safety and efficacy profiles that are significantly superior to those achieved by the use of naturally occurring genes. This procedure may contribute considerably to fulfilling standards set by regulatory authorities. This book provides an overview of the current advances in the field of gene therapy and the methods that are being successfully applied in the manufacture of gene therapeutic products, and hopefully will stimulate further progress and advancement in this field to meet the ever-increasing demands.

Somatic Gene Therapy

Somatic Gene Therapy PDF Author: P.L. Chang
Publisher: CRC Press
ISBN: 1351093665
Category : Science
Languages : en
Pages : 242

Get Book Here

Book Description
As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy.

Regulatory Aspects of Gene Therapy and Cell Therapy Products

Regulatory Aspects of Gene Therapy and Cell Therapy Products PDF Author: Maria Cristina Galli
Publisher: Springer
ISBN: 3319186183
Category : Medical
Languages : en
Pages : 235

Get Book Here

Book Description
This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127

Get Book Here

Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.