Titlebook: Gene Therapy for Neurological Disorders; Methods and Protocol Fredric P. Manfredsson Book 2016 Springer Science+Business Media New York 201

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Altering Tropism of rAAV by Directed Evolution of virions for which each particular capsid variant is matched with the particular capsid gene encoding it; recovery of capsid gene sequences from target tissue after systemic administration. Prevalent variants are then analyzed and evaluated.

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Norbert Clauer,Sambhu Chaudhuriiverse diseases including hemophilia B and Leber’s congenital amaurosis. In addition to rAAV’s high efficiency of transduction and the capacity for long-term transgene expression, the safety profile of rAAV remains unsoiled in humans with no deleterious vector-related consequences observed thus far.

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