Engineering targeted viral vectors for gene therapy
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Updated: Jul 14, 2026

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants
Published on: October 18, 2022
Reinhard Waehler1, Stephen J Russell, David T Curiel
1Division of Human Gene Therapy, 502 Biomedical Research Building II, 901 19th Street, South Birmingham, Alabama 35294-2172, USA.
Abstract:
To achieve therapeutic success, transfer vehicles for gene therapy must be capable of transducing target cells while avoiding impact on non-target cells. Despite the high transduction efficiency of viral vectors, their tropism frequently does not match the therapeutic need. In the past, this lack of appropriate targeting allowed only partial exploitation of the great potential of gene therapy. Substantial progress in modifying viral vectors using diverse techniques now allows targeting to many cell types in vitro. Although important challenges remain for in vivo applications, the first clinical trials with targeted vectors have already begun to take place.
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