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Updated: May 25, 2026
09:20
Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants
Published on: October 18, 2022
The AAV vector toolkit: poised at the clinical crossroads
Aravind Asokan1, David V Schaffer, R Jude Samulski
1Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599-7352, USA. aravind@med.unc.edu
Summary
Adeno-associated virus (AAV) vectors show promise for gene therapy, with engineered strains overcoming challenges like immune responses and improving efficiency in clinical trials for genetic diseases.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- Naturally occurring and engineered adeno-associated virus (AAV) strains form a versatile toolkit for gene delivery.
- Preclinical success and early clinical trials demonstrate AAV's potential for treating genetic diseases.
Purpose of the Study:
- To review preclinical studies of AAV vectors in large animal models.
- To provide an update on novel AAV vector candidates for clinical translation.
Main Methods:
- Exploration of naturally occurring AAV isolates across species.
- Application of molecular genetics for engineering AAV strains.
- Evaluation of AAV vector performance in preclinical animal models and human clinical trials.
Main Results:
- First-generation AAV vectors face challenges including cross-species tropism variation, pre-existing immunity, and dose-dependent toxicity.
- Second-generation AAV vectors have been engineered to address these limitations.
- Early clinical trials show promise but highlight areas for improvement.
Conclusions:
- Ongoing development of AAV vectors is crucial for advancing gene therapy.
- Second-generation AAV vectors offer improved safety and efficacy profiles.
- Further research in large animal models and clinical translation is essential for optimizing AAV-based therapies.
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