Lecture

Choosing a Viral Vector and Delivery Method

Description

This lecture covers the selection of viral vectors for gene therapy, focusing on factors such as genomic integration, cell types transduced, transcript length, vector design, viral genome, and immune reactions. Specific examples are provided, including the use of adeno-associated virus (AAV) vectors for spinal muscular atrophy type 1 (SMA1) therapy and different routes of administration for AAV vectors in the central nervous system.

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