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

15 Jan 2024

Abstract excerpt

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 disorders. Owing to the intricacy of the human CNS and the blood–brain barrier, it is difficult to deliver genes,...

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PMID: 38256124
DOI: 10.3390/ijms25021050

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