A versatile cell line for establishing potency of cell type-specific AAV transgenes

A versatile cell line for establishing potency of cell type-specific AAV transgenes

Potency assays are essential for adeno-associated virus (AAV) therapy development, allowing simple and quantitative assessments of therapeutic activity, target engagement, vector stability, and activity per batch. However, cell culture-based AAV potency assays are generally hampered by two limitatio...

Full description

Saved in:
Bibliographic Details
Main Authors: Refugio A. Martinez, Yemeserach M. Bishaw, Meagan A. Quinlan, Erin L. Groce, Bryan B. Gore, Ed S. Lein, John K. Mich, Boaz P. Levi
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
AAVR
CRISPRa
in vitro potency assay
HEK293T cells
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124002171
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items

Similar Items