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Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of gene transfer agents in the context of their clinical use requires investigators from a wide variety of backgrounds to have an understanding — or at least an appreciation of — the regulatory environment and constraints that affect vector design, manufacturing, pre-clinical testing, and clinical use, with an emphasis on patient protection. In Adeno-Associated Virus: Methods and Protocols, experts from the United States and Europe have contributed current knowledge of this multi-dimensional field relating to the biology of AAV, rAAV vector design, vector manufacturing and product testing, performance of rAAV vectors in major organs, rAAV-related immunological issues, design of animal and clinical studies, and clinical experience. Written in the successful Methods in Molecular Biology™ series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and accessible, Adeno-Associated Virus: Methods and Protocols provides a complete and comprehensive understanding of this multi-disciplinary and rapidly progressing field.

Methods in Molecular Biology, Methods and Protocols

Springer eBooks

XIII, 462 pages 43 illustrations, 20 illustrations in color : online resource.

Adeno-Associated Virus : Methods and Protocols / edited by Richard O. Snyder, Philippe Moullier.

In Gene Therapy Protocols, Volumes 1 & 2, internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors (Volume 1) and Design and Characterization of Gene Transfer Vectors (Volume 2). The field of gene therapy has undergone remarkable advances, promising to impact human healthcare significantly in the twenty-first century. Today’s technologies can deliver genetic material safely and effectively to cells to slow or halt the progression of disease, and to help repair or regenerate damaged or lost tissues. In this first volume of Gene Therapy Protocols: Production and In Vivo Applications of Gene Transfer Vectors, readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for critical applications in stem cell biology, cancer, diabetes, HIV and tissue engineering. This first volume of the new and completely revised third edition of Gene Therapy Protocols will prove a necessary tool for graduate students and postdoctoral fellows and invaluable to basic and clinical researchers in both industry and academia.

Methods in Molecular Biology™

Gene Therapy Protocols : Production and In Vivo Applications of Gene Transfer Vectors / by Joseph M. Le Doux.

Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular Biology™ series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.

RNA Therapeutics : Function, Design, and Delivery / edited by Mouldy Sioud.

ScienceDirect Subject Collections - Biochemistry, Genetics and Molecular Biology

Trends in molecular medicine

Landkarte der gene : das genom-projekt / Jerry E. Bishop, Michael Waldholz.

Gene therapy / Health Council of the Netherlands, Committee on Gene Therapy.

His brother's keeper : a story from the edge of medicine / Jonathan Weiner.

Peter Evans looks at problems occurring in clinical trials in the use of gene therapy to treat serious diseases. Chris Higgins of Hammersmith Hospital talks about his work using gene therapy to treat Cystic Fibrosis. David Kerr of Birmingham University describes his research into how gene therapy might be used to treat cancer.

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Adeno-Associated Virus : Methods and Protocols / edited by Richard O. Snyder, Philippe Moullier.

Gene Therapy Protocols : Production and In Vivo Applications of Gene Transfer Vectors / by Joseph M. Le Doux.

RNA Therapeutics : Function, Design, and Delivery / edited by Mouldy Sioud.

Trends in molecular medicine

Landkarte der gene : das genom-projekt / Jerry E. Bishop, Michael Waldholz.

Gene therapy / Health Council of the Netherlands, Committee on Gene Therapy.

His brother's keeper : a story from the edge of medicine / Jonathan Weiner.

Fixing genes : fixing genes.

Gene therapy and regulation

Gene therapy and regulation

Stay in the know

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More filters

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Adeno-Associated Virus : Methods and Protocols / edited by Richard O. Snyder, Philippe Moullier.

Gene Therapy Protocols : Production and In Vivo Applications of Gene Transfer Vectors / by Joseph M. Le Doux.

RNA Therapeutics : Function, Design, and Delivery / edited by Mouldy Sioud.

Trends in molecular medicine

Landkarte der gene : das genom-projekt / Jerry E. Bishop, Michael Waldholz.

Gene therapy / Health Council of the Netherlands, Committee on Gene Therapy.

His brother's keeper : a story from the edge of medicine / Jonathan Weiner.

Fixing genes : fixing genes.

Gene therapy and regulation

Gene therapy and regulation

Stay in the know

Wellcome collectionWellcome Collection

Finding us:

Opening times:

Today's opening times

Wellcome collection