The Gene Therapy Toolbox: AAV

Date/Time: Monday, October 5, 2020 - 12:00 PM – 1:00 PM
Track: Interactive Lunch Workshop
Room: San Gabriel B
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Description:

Following the approval of the first gene therapy for a neurological disease, a growing number of clinical trials predicts additional approvals in the next decade, most using AAV as a vehicle. Neurologists should familiarize with the status of the field for these “one and done” therapies.

Objective(s):

  • To understand the biology of AAV, repertoire of capsids, species specificity and immune responses.

  • To learn the different AAV-driven therapeutic strategies available

  • To identify currently approved and emerging AAV-based therapies for neurological diseases.