Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays
Vectors based on adeno-associated viruses (AAVs) are promising therapeutic modalities used in gene therapy. Robust cell-based assays that demonstrate and quantify the potency of AAV vectors in expressing their transgene are needed for clinical development. However, many AAV clinical serotypes poorly...
Saved in:
Main Authors: | James Zengel, Emma S. Esterman, Anitha Ponnuswami, Nicholas R. Wall, Jan E. Carette |
---|---|
Format: | Article |
Language: | English |
Published: |
Elsevier
2025-03-01
|
Series: | Molecular Therapy: Methods & Clinical Development |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050125000117 |
Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution
by: Florian Dunker-Seidler, et al.
Published: (2025-03-01) -
Probing aspects of extracellular vesicle associated AAV allows increased vector yield and insight into its transduction and immune-evasive properties
by: Ming Cheng, et al.
Published: (2025-03-01) -
Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates
by: Mengping Liu, et al.
Published: (2025-03-01) -
AAV-based vectors for human diseases modeling in laboratory animals
by: Timur I. Aliev, et al.
Published: (2025-02-01) -
Research Progress of ROS-Mitochondrial Pathway on Cardiovascular Protection of Tea Polyphenols
by: Tingting DENG, et al.
Published: (2025-02-01)