Prenatal Gene Therapy: Concepts, Methods, and Protocols by Charles Coutelle MD, DSc, Simon N. Waddington PhD (auth.),

By Charles Coutelle MD, DSc, Simon N. Waddington PhD (auth.), Charles Coutelle, Simon N. Waddington (eds.)

The rising box of prenatal gene remedy is based on medical and technical advances in fetal drugs, molecular biology and gene remedy. This preclinical study topic goals at using gene remedy while pregnant for the prevention of human ailments because of early onset congenital or gestation comparable stipulations. In Prenatal Gene treatment: techniques, equipment and Protocols, professional researchers within the box aspect a number of the protocols that are now frequent to review gene treatment, fetal medication and clinical ethics. those comprise distinct protocols for vector construction, for breeding and husbandry of the animal types, for the surgeries of gene supply in huge and small animals and for the tools of gene move research. Written within the hugely winning Methods in Molecular Biology™ sequence structure, chapters comprise introductions to their respective themes, lists of the required fabrics and reagents, step by step, without difficulty reproducible laboratory protocols, and key pointers on troubleshooting and fending off recognized pitfalls.

Thorough and intuitive, Prenatal Gene remedy: suggestions, equipment and Protocols seeks to assist scientists within the extra learn of prenatal ailment and gene remedy tasks past the scope of fetal medicine.

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A phase I trial targeting the liver through intra-arterial adenovirus injection ended with low level gene transfer and a fatal immune reaction in one of the 17 patients (131, 132). Subsequent investigation in small animals focused on less immunogenic vectors and showed that long-term correction of the metabolic defect in OTC deficiency could be achieved using a helper-dependent adenovirus vector (133) and AAV (134). Because of the difficulties with postnatal OTC deficiency gene therapy and the severity and very early onset of the complete form, fetal gene therapy may be a good approach.

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