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Cover -- Half Title -- Title Page -- Copyright Page -- Table of Contents -- Acknowledgements -- Series Preface -- Introduction -- Part I Genetics - General -- 1 Julian Kinderlerer and Diane Longley (1998), 'Human Genetics: The New Panacea?', Modern Law Review, 61, pp. 603-20 -- 2 Julia Black (1998), 'Regulation as Facilitation: Negotiating the Genetic Revolution', Modern Law Review, 61, pp. 621-60 -- 3 Darryl Macer (1991), 'Whose Genome Project?', Bioethics, 5, pp. 183-211 -- 4 Sheila A.M. McLean (2001), 'The Gene Genie: Good Fairy or Wicked Witch?', Studies in History and Philosophy of Biological and Biomedical Sciences, 32, pp.723-39 -- 5 John A. Robertson (2003), 'Procreative Liberty in the Era of Genomics', American Journal of Law and Medicine, 29, pp. 439-87 -- 6 Susan M. Wolf (1995), 'Beyond "Genetic Discrimination": Toward the Broader Harm of Geneticism', Journal of Law, Medicine and Ethics, 23, pp. 345-53 -- 7 Deborah Hellman (2003), 'What Makes Genetic Discrimination Exceptional?', American Journal of Law and Medicine, 29, pp. 77-116 -- 8 Dean Bell and Belinda Bennett (2001), 'Genetic Secrets and the Family', Medical Law Review, 9, pp. 130-61 -- 9 Lawrence O. Gostin (1995), 'Genetic Privacy', Journal of Law, Medicine and Ethics, 23, pp. 320-30 -- 10 Graeme T. Laurie (2001), 'Challenging Medical-Legal Norms: The Role of Autonomy, Confidentiality, and Privacy in Protecting Individual and Familial Group Rights in Genetic Information', Journal of Legal Medicine, 22, pp. 1-54 -- 11 Philippa Gannon and Charlotte Villiers (1999), 'Genetic Testing and Employee Protection', Medical Law International, 4, pp. 39-57 -- 12 Allen Buchanan, Andrea Califano, Jeffrey Kahn, Elizabeth McPherson, John Robertson and Baruch Brody (2002), 'Pharmacogenetics: Ethical Issues and Policy Options', Kennedy Institute of Ethics Journal, 12, pp. 1-15

Cover -- Half Title Page -- Title Page -- Copyright Page -- Declaration -- About the Editor -- Table of Contents -- List of Contributors -- List of Abbreviations -- Preface -- Chapter 1 Gene Therapy: Progress and Predictions -- Abstract -- Introduction -- Gene Therapy Remains A Delivery Challenge -- Replacement Gene Therapy Using Bone Marrow Transplantation -- Direct Injection of Adeno-Associated Virus Vectors For Gene Replacement Therapy -- Gene Therapy to Treat Cancer And Infectious Disease -- Gene Therapy as a Different Formulation of a Conventional Medicine -- Where Will Gene Therapy Be In Another 25 Years? -- References -- Chapter 2 Advances In Non-Viral DNA Vectors For Gene Therapy -- Abstract -- Introduction to Gene Therapy -- Challenges of Using Plasmid Vectors For Gene Therapy -- Improvements to Plasmid Vectors -- Development of Minicircles and Minivectors -- Therapeutic Needs Best Addressed by Minimized Vectors -- Concluding Remarks: Moving Minimized Non-Viral DNA Vectors Into The Clinic -- Acknowledgments -- Author Contributions -- References -- Chapter 3 Gene Delivery Technologies For Efficient Genome Editing: Applications In Gene Therapy -- Abstract -- Introduction -- Viral-Based Vectors -- Non-Viral-Based Vectors -- Acknowledgements -- References -- Chapter 4 Alpharetroviral Vectors: From A Cancer-Causing Agent To A Useful Tool For Human Gene Therapy -- Abstract -- Introduction -- History of (Alpha-) Retroviruses -- From The Virus To The Vector -- On The Road To Clinical Applicability: Production Perspectives of Alpharetroviral Vectors -- From Bench To Bedside: Regulatory Requirements For Clinical Translation -- Potential Future Clinical Applications of Alpharetroviral Sin Vectors -- Conclusions And Outlook -- Acknowledgments -- References And Notes

Bridging brain barriers for gene therapy
Reflecting on the challenges in treating brain diseases, this article explores ways to transduce the blood-brain barrier as well as the critical role of tanycytes as a target for gene therapy vectors. It is vital that the precious central nervous system is protected from exogenous harmful factors that can damage it. As an innate helmet, the skull protects the brain from mechanical trauma. The blood-brain barrier, on the other hand, serves as a much less conspicuous shield that prevents molecular injury from taking place.

This book addresses the latest advances in viral and non-viral vectors, novel targets and methods for the treatment of autoimmune and inflammatory disease with a main focus in pre-clinical testing with up to date clinical trials. The first section of the book deals with current research approaches for treating rheumatoid arthritis, osteoarthritis, diabetes, Crohn's disease, multiple sclerosis, Sjögrens syndrome, cystic fibrosis, myositis and lupus. The second section is devoted to cell delivery and vector development including plasmids, regulated promoters, adenovirus gutted vectors and adeno-associated vectors. All chapters are summarised by leaders in these fields. A must read for post graduate students, clinical and non-clinical researchers in the disciplines of immunology, molecular medicine, pharmacology, gene and cell-mediated therapy.

The "National Guidelines for Gene Therapy Product (GTP) Development and Clinical Trials" prepared by the Indian Council of Medical Research and Department of Biotechnology in 2019 came as a welcome step in the process of regulation of gene therapy research, as there was a lack of Indian guidelines earlier specific to gene therapy. Indian researchers have taken their step in setting the path of gene therapy research, and this guideline serves to provide the standards starting from its development up to translation to new drug including the ethical, scientific, and regulatory requirements to be followed during the conduct of trial. The Indian guidelines were framed with reference to United States-Food and Drug Administration and European Union guidelines on gene therapy. It is the responsibility of all the stakeholders involved in the development of GTP to adhere to the national guidelines. This review provides an outline of the Indian regulatory guidelines on GTP.

On May 11, 1993, the National Academy of Sciences hosted a workshop convened to address the scientific, medical, and social issues arising from attempts to enlist gene therapy experimentation in the fight against AIDS. The 100 participants represented a cross-section of federal governmental, industrial, academic, and research-practitioner expertise. I was invited presumably because I am a member of the Recombinant DNA Advisory Committee (RAC), an arm of the National Institutes of Health, which oversees (in effect, licenses) human gene therapy protocols, and because I have recently published reports bearing on that policy process (Carmen, 1992, 1993).