Title Prenatal gene therapy : concepts, methods, and protocols / edited by Charles Coutelle, Simon N. Waddington

Published Totowa, NJ : Humana Press, ©2012

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Description 1 online resource (x, 392 pages) : illustrations (some color) Series Methods in molecular biology, 1940-6029 ; 891 Methods in molecular biology (Clifton, N.J.) ; v. 891. 1064-3745 Contents The concept of prenatal gene therapy / Charles Coutelle and Simon N. Waddington -- Candidate diseases for prenatal gene therapy / Anna L. David and Simon N. Waddington -- Vector systems for prenatal gene therapy: choosing vectors for different applications / Charles Coutelle and Simon N. Waddington -- Vector systems for prenatal gene therapy: principles of adenovirus design and production / Raul Alba, Andrew H. Baker, and Stuart A. Nicklin -- Vector systems for prenatal gene therapy: principles of retrovirus vector design and production / Steven J. Howe and Anil Chandrashekran -- Vector systems for prenatal gene therapy: principles of adeno-associated virus vector design and production / Christopher J. Binny and Amit C. Nathwani -- Vector systems for prenatal gene therapy: principles of non-viral vector design and production / Suet Ping Wong, Orestis Argyros, and Richard P. Harbottle -- Use of manipulated stem cells for prenatal therapy / Jessica L. Roybal, Pablo Laje, Jesse D. Vrecenak, and Alan W. Flake -- Animal models for prenatal gene therapy: choosing the right model / Vedanta Mehta, Donald Peebles, and Anna L. David -- Animal models for prenatal gene therapy: rodent models for prenatal gene therapy / Jessica L. Roybal, Masayuki Endo, Suzanne M.K. Buckley, Bronwen R. Herbert, Simon N. Waddington, and Alan W. Flake -- Animal models for prenatal gene therapy: the sheep model / Khalil N. Abi-Nader, Michael Boyd, Alan W. Flake, Vedanta Mehta, Donald Peebles, and Anna L. David -- Animal models for prenatal gene therapy: the nonhuman primate model / Citra N. Mattar, Arijit Biswas, Mahesh Choolani, and Jerry K.Y. Chan -- Choice of surrogate and physiological markers for prenatal gene therapy / Juliette M.K.M. Delhove, Ahad A. Rahim, Tristan R. McKay, Simon N. Waddington, and Suzanne M.K. Buckley -- Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application / Vedanta Mehta, Khalil N. Abi-Nader, David Carr, Jacqueline Wallace, Charles Coutelle, Simon N. Waddington, Donald Peebles, and Anna L. David -- Monitoring for potential adverse effects of prenatal gene therapy: mouse models for developmental aberrations and inadvertent germ line transmission / Charles Coutelle, Simon N. Waddington, and Michael Themis -- Monitoring for potential adverse effects of prenatal gene therapy: genotoxicity analysis in vitro and on small animal models ex vivo and in vivo / Michael Themis -- Risks, benefi ts and ethical, legal, and societal considerations for translation of prenatal gene therapy to human application / Charles Coutelle and Richard Ashcroft Summary The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly 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 laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls. Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine Analysis Life sciences Human genetics Gene therapy Animal genetics gentherapie genomica genomics prenatale zorg prenatal care prenatale ontwikkeling prenatal development humane genetica Medicine (General) Geneeskunde (algemeen) Bibliography Includes bibliographical references and index Notes English Online resource; title from PDF title page (Springer Protocols, viewed Oct. 12, 2012) Subject Gene therapy. Fetus -- Diseases -- Treatment Therapeutics. Therapeutics Investigative Techniques Publication Formats Publication Characteristics Analytical, Diagnostic and Therapeutic Techniques and Equipment Fetal Therapies Models, Animal Laboratory Manuals Genetic Therapy treating (health care function) Therapeutics Fetus -- Diseases -- Treatment Gene therapy Genre/Form Laboratory manuals. Manuels de laboratoire. Form Electronic book Author Coutelle, Charles, 1939- Waddington, Simon N. ISBN 9781617798733 1617798738 161779872X 9781617798726

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