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

Santoscoy, Miguel C; Espinoza, Paula; De La Cruz, Demitri; Mahamdeh, Mohammed; Starr, Jacqueline R; Patel, Nikita; Maguire, Casey A

Santoscoy, Miguel C; Espinoza, Paula; De La Cruz, Demitri; Mahamdeh, Mohammed; Starr, Jacqueline R; Patel, Nikita; Maguire, Casey A.

Afiliación

Santoscoy MC; Harvard Medical School, Boston, MA, USA.
Espinoza P; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
De La Cruz D; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.
Mahamdeh M; Harvard Medical School, Boston, MA, USA.
Starr JR; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
Patel N; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA, USA.
Maguire CA; Harvard Medical School, Boston, MA, USA.

Mol Ther Methods Clin Dev ; 29: 532-540, 2023 Jun 08.

Article en En | MEDLINE | ID: mdl-37359416

Palabras clave

AAV; adeno-associated virus vector; brain; cell-type specific promoter; cholinergic neurons; gene delivery; gene therapy; striatum; target gene expression; transduction

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