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Luk H. Vandenberghe, PhD - "Deciphering AAV, Reprogramming Pharmacology"

Grousbeck Family Chair in Gene Therapy - Associate Professor, Dept. of Ophthalmology, Harvard - Associate Member, The Broad Institute of Harvard and MIT - Director, Grousbeck Gene Therapy Center - Boston, MA, USA
When Mar 19, 2020
from 12:00 PM to 01:15 PM
Where Tigem, Auditorium Vesuvius
Contact Name
Contact Phone 08119230659
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Abstract
Therapeutic gene transfer is a novel drug modality that can transform the life of patients. AAV has emerged as a powerful in vivo gene transfer vector based on a naturally occurring virus with validaton in the lab, clinic, and more recently in the marketplace. Remarkably, AAV remains poorly understood from a mechanistic level which limits our ability to engineer tropism, host response, or manufacturing properties which may limit applications. Here, we describe a novel approach to interrogate the structure-function relationship of AAV in a high-throughput manner in a way that can inform rational design of AAV in clinically relevant ways (e.g. across species barriers).

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