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This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease
Viral vectors are superior tools for gene therapy and as a genetic vaccine platform because viruses have evolved to efficiently infect and transfer their genomes to cells. Several impressive successes in viral vector-based gene therapies have been reported in humans, including restoration of vision in patients with Leber’s congenital amaurosis by retinal gene transfer and cures for severe immune deficiencies by gene transfer to hematopoietic stem cells. However, the mammalian immune system has evolved in parallel to fend off invading pathogens such as viruses. Innate and antigen-specific adaptive immune responses against viral vectors and therapeutic transgene products pose serious hurdles...
Hepatitis, the leading indication for liver transplantation, is a straight-forward disease when it comes to diagnosis: it is a disease of pathology and is diagnosed by liver biopsy, with the result being either positive or negative. There is only one effective treatment: interferons. Nonetheless, new interferons are appearing on the market and Drs Foster and Goldin examine the use of each of these in the management of chronically infected patients. The authors have written a succinct, highly illustrated text for all those interested in the management of viral hepatitis.
Emily B. Baran offers a gripping history of how a small, American-based religious community, the Jehovah's Witnesses, found its way into the Soviet Union after World War II, survived decades of brutal persecution, and emerged as one of the region's fastest growing religions after the Soviet Union's collapse in 1991. In telling the story of this often misunderstood faith, Baran explores the shifting boundaries of religious dissent, non-conformity, and human rights in the Soviet Union and its successor states. Soviet Jehovah's Witnesses are a fascinating case study of dissent beyond urban, intellectual nonconformists. Witnesses, who were generally rural, poorly educated, and utterly marginaliz...
Gene therapy has generated enormous expectations as it holds a great promise, namely to revolutionize clinical and molecular medicine. This is because the successful introduction of a gene sequence in somatic sells followed by long-term expression at therapeutic levels can fundamentally correct a variety of human disease such as inherited and acquired disorders, cancer and AIDS. The proceedings of the first ASI on Gene Therapy that took place from August 17-28, 1997 on the island of Spetsai, Greece, summarizes recent advances in the field as presented elegantly by the intemationally recognized experts that served as faculty at the meeting. Their contributions constitute brief but comprehensi...
Major topics covered include photoreceptor proteins, phototransduction calcium-binding proteins and calcium measurement in photoreceptor cells, enzymes of the visual cycle, posttranslational and chemical modifications, analysis of animal models of retinal diseases. Inherited retinal disease; from the defective gene to its function and repair. This volume and its companion Volume 315 include newly developed methods to study vertebrate phototransduction and the visual cycle.The critically acclaimed laboratory standard for more than forty years, Methods in Enzymology is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. Now with more than 300 volumes (all of them still in print), the series contains much material still relevant today--truly an essential publication for researchers in all fields of life sciences.
Several major developments have occurred since the last edition of Handbook of Obesity: Clinical Applications, including new clinical trials, discoveries related to drug use, and greater understanding of the benefits of weight loss in obese patients. Now in its fourth edition, this volume continues to offer unparalleled depth and breadth of coverag
The European Court of Human Rights, the Inter-American Court of Human Rights, and the African Court on Human and Peoples' Rights are three supranational jurisdictions that protect human rights. This book is the first comprehensive study to compare the three regional courts. It also considers how they operate as parts of a greater whole.
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