Year: 2024
Type: article
Abstract: AAV vectors continue to be the most promising gene delivery vehicle for treating rare genetic diseases through gene therapy. Understanding vector inconsistencies during the manufacturing process is vital to define batch-to-batch differences, and predicting their efficacies and safety profiles. Although AAV vectors manufactured for clinical use are rigorously tested by several analytical methods, these assays are still not able to provide comprehensive insights into a vector’s composition, nor address how or why heterogeneity in vectors emerge. With the power of next-generation sequencing methods, understanding AAV vector composition and why certain designs fail to provide expected potencies can be unlocked.
Source: Cell and Gene Therapy Insights
Authors: Ngoc Tam Tran, Phillip WL Tai
Institution: University of Massachusetts Chan Medical School
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Funders: Biogen, BioMarin Pharmaceutical, REGENXBIO
OpenAlex ID: W4391531730