Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

Genetic disorders of the central nervous system (CNS) comprise a significant portion of disability in both children and adults. Several preclinical animal models have shown effective adeno-associated virus (AAV) mediated gene transfer for either treatment or prevention of autosomal recessive genetic...

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Bibliographic Details
Main Authors: Rrita Daci, Terence R. Flotte
Format: Article
Language:English
Published: MDPI AG 2024-01-01
Series:International Journal of Molecular Sciences
Subjects:
gene therapy
adeno-associated virus vector
AAV
gene delivery
inherited disorders
Online Access:https://www.mdpi.com/1422-0067/25/2/1050

Internet

https://www.mdpi.com/1422-0067/25/2/1050

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