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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Gene Therapy for Neurological Disorders978-1-4939-3271-9Series ISSN 1064-3745 Series E-ISSN 1940-6029

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Intraparenchymal Stereotaxic Delivery of rAAV and Special Considerations in Vector Handlingpowerful tool that provides the ability to manipulate gene expression in specific regions, or even specific cell types in the brain. Here, we describe the proper handling and stereotaxic delivery of recombinant adeno-associated virus to various neuroanatomical structures of the rodent brain.

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Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous Systeme practical application of viral or non-viral gene therapy is not as straightforward as it may seem. All too often investigators see their experiments fail due to low-quality third-party vectors or due to a lack of knowledge regarding the proper use of these tools. For example, researchers often fin

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Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectorsiverse 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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