An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction

An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction

Adeno-associated virus (AAV) vectors are currently the most efficient option for intracranial gene therapies to treat neurodegenerative disease. Increased efficacy and safety will depend upon robust and specific expression of therapeutic genes into target cell-types within the human brain. In this s...

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Bibliographic Details
Main Authors: Miguel C. Santoscoy, Paula Espinoza, Demitri De La Cruz, Mohammed Mahamdeh, Jacqueline R. Starr, Nikita Patel, Casey A. Maguire
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
striatum
cholinergic neurons
AAV
cell-type specific promoter
target gene expression
adeno-associated virus vector
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000633

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http://www.sciencedirect.com/science/article/pii/S2329050123000633

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