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A Guide to Human Gene Therapy
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-clini...
Tolerating Factor VIII: Novel Strategies to Prevent and Reverse Anti-FVIII Inhibitors
The article processing charges (APCs) for some articles in this collection were partly financed by the Henry M. Jackson Foundation for the Advancement of Military Medicine, Maryland, USA (HJF), with funds which were originally granted by Grifols S.A, Barcelona, Spain (Grifols). Neither HJF nor Grifols influenced the content of any article in this collection.
Gene Therapy Immunology
Summarises and reviews the important field of genetic therapy with respect to the latest immunological advances in the lab and clinic Unique treatment of immunology and immunotherapy in gene - approached from a vector and target organ point of view rather than from the perspective a specific diseases Broad appeal - applicable for immunology and genetics / gene therapy, recombinant DNA studies, transplantation, virology, cancer research and tumor research
The Patient as Victim and Vector
This volume is jointly written by four authors at the University of Utah with expertise in bioethics, health law, and infectious disease. In collaboration they attempt to develop a normative framework sensitive to situations of disease transmission- situations in which the patient is not only a victim but a vector; i.e. vulnerable to disease but also a threat to others.
Designing Our Descendants
The Human Genome Project, discoveries in molecular biology, and new reproductive technologies have advanced our understanding of how genetic science may be used to treat persons with genetic disorders. Greater knowledge may also make possible genetic interventions to "enhance" normal human characteristics, such as height, hair or eye color, strength, or memory, as well as the transmittal of such modifications to future generations. The prospect of inheritable genetic modifications, or IGMs, whether for therapeutic or enhancement purposes, raises complex scientific, ethical, and regulatory issues. Designing Our Descendants presents twenty essays by physicians, scientists, philosophers, theolo...
The Immunological Barriers to Regenerative Medicine
This volume offers an analysis of the scale and nature of the immunological issues facing regenerative medicine, drawing on the expertise of laboratories around the world who have taken up the challenge of applying their expertise in immunology to the vagaries of stem cell biology. In Part I, we explore the extent to which the principles of allograft rejection, learned over several decades from our experiences of whole organ transplantation, apply within the unique context of cell replacement therapy. Part II discusses various innovative ways of addressing the issues of immunogenicity, while, in Part III, we focus exclusively on the induction of immunological tolerance through a variety of novel approaches. It is our hope that this systematic analysis of the current state of the field will galvanise efforts to solve an issue which has so far remained intractable.
Fast Facts: Gene Therapy
Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments
The Patient as Victim and Vector
This volume is jointly written by four authors at the University of Utah with expertise in bioethics, health law, and infectious disease. In collaboration they attempt to develop a normative framework sensitive to situations of disease transmission- situations in which the patient is not only a victim but a vector; i.e. vulnerable to disease but also a threat to others. This reissue includes a new preface exploring the implications of the Covid-19 pandemic.
Gene Delivery to Mammalian Cells
The efficiency of delivering DNA into mammalian cells has increased t- mendously since DEAE dextran was first shown to be capable of enhancing transfer of RNA into mammalian cells in culture. Not only have other chemical methods been developed and refined, but also very efficient physical and viral delivery methods have been established. The technique of introducing DNA into cells has developed from transfecting tissue culture cells to delivering DNA to specific cell types and organs in vivo. Moreover, two important areas of biology—assessment of gene function and gene therapy—require succe- ful DNA delivery to cells, driving the practical need to increase the efficiency and efficacy of ...
AAV Gene Therapy: Immunology and Immunotherapeutics
Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme
