Development of a one-step RT-ddPCR method to determine the expression and potency of AAV vectors

Robust assays to quantify adeno-associated virus (AAV) vector expression and potency are essential for gene therapy development. These assays inform the efficacy, safety, and pharmacodynamic profiles of AAV development candidates. Additionally, for gene downregulation strategies such as RNAi, knockd...

Full description

Bibliographic Details
Main Authors: Pete Clarner, Shukkwan K. Lau, Twinkle Chowdhury, Edward Guilmette, Patrick Trapa, Shih-Ching Lo, Shen Shen
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000875