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Adeno-Associated Virus Vector for Central Nervous System Gene Therapy.

Trends in molecular medicine - 1 Jun 2021

Zhu Danqing, Schieferecke Adam J, Lopez Paola A, Schaffer David V

Abstract excerpt

The past several years have witnessed significant advances in the development of therapeutic gene delivery for neurological disorders of the central nervous system (CNS). In particular, genome-wide sequencing analysis has deepened our understanding of mutations that underlie many monogenic disorders, which in turn has contributed to clinical advances involving adeno-associated virus (AAV) vector delivery of...

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PMID: 33895085
DOI: 10.1016/j.molmed.2021.03.010

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