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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Detalles Bibliográficos
Autores principales: Santoscoy, Miguel C., Espinoza, Paula, De La Cruz, Demitri, Mahamdeh, Mohammed, Starr, Jacqueline R., Patel, Nikita, Maguire, Casey A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285237/
https://www.ncbi.nlm.nih.gov/pubmed/37359416
http://dx.doi.org/10.1016/j.omtm.2023.05.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285237/
https://www.ncbi.nlm.nih.gov/pubmed/37359416
http://dx.doi.org/10.1016/j.omtm.2023.05.001

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