The Gene Therapy Toolbox: AAV

Date/Time: Wednesday, October 7, 2020 - 12:30 PM – 1:45 PM
Track: Interactive Workshop
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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.


  • 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.


Introduction to AAV as a Delivery Vehicle for Gene Therapy in Neurology

Lessons from Completed Ongoing AAV Gene Therapy Studies in Parkinson's Disease

Therapeutic Approaches using AAV: Gene Transfer, Silencing and Editing