Adeno-associated virus vector as a platform for gene therapy delivery
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Updated: Jan 30, 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
Dan Wang1,2,3, Phillip W L Tai1,2,3, Guangping Gao4,5,6
1Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Abstract:
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. Continued study of AAV biology and increased understanding of the associated therapeutic challenges and limitations will build the foundation for future clinical success.
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A vector multiplied by a scalar value is called scalar multiplication. The result obtained is a new vector with a different magnitude. If the scalar is positive, the direction of the vector remains the same, but if it is negative, the direction of the vector is reversed. For example, the product of the mass and velocity yields the momentum.

