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Delivering AAV to the Central Nervous and Sensory Systems.

ABSTRACT: As gene therapy enters mainstream medicine, it is more important than ever to have a grasp of exactly how to leverage it for maximum benefit. The development of new targeting strategies and tools makes treating patients with genetic diseases possible. Many Mendelian disorders are amenable to gene replacement or correction. These often affect post-mitotic tissues, meaning that a single stably expressing therapy can be applied. Recent years have seen the development of a large number of novel viral vectors for delivering specific therapies. These new vectors - predominately recombinant adeno-associated virus (AAV) variants - target nervous tissues with differing efficiencies. This review gives an overview of current gene therapies in the brain, ear, and eye, and describes the optimal approaches, depending on cell type and transgene. Overall, this work aims to serve as a primer for gene therapy in the central nervous and sensory systems.

SUBMITTER: Peters CW

PROVIDER: S-EPMC9302199 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature

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Delivering AAV to the Central Nervous and Sensory Systems.

Peters Cole W CW Maguire Casey A CA Hanlon Killian S KS

Trends in pharmacological sciences 20210413 6

As gene therapy enters mainstream medicine, it is more important than ever to have a grasp of exactly how to leverage it for maximum benefit. The development of new targeting strategies and tools makes treating patients with genetic diseases possible. Many Mendelian disorders are amenable to gene replacement or correction. These often affect post-mitotic tissues, meaning that a single stably expressing therapy can be applied. Recent years have seen the development of a large number of novel vira ...[more]

PMID: 33863599

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