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Development of adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model

Viral vector gene therapy has immense promise for treating central nervous system (CNS) disorders. Although adeno-associated virus vectors (AAVs) have had success, their small packaging capacity limits their utility to treat the root cause of many CNS disorders. Adenoviral vectors (Ad) have tremendo...

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Uloženo v:
Hlavní autoři: Emre Kul, Uchechi Okoroafor, Amanda Dougherty, Lauren Palkovic, Hao Li, Paula Valiño-Ramos, Leah Aberman, Samuel M. Young, Jr.
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2024-06-01
Edice:Molecular Therapy: Methods & Clinical Development
Témata:
viral vector gene therapy
retrograde transduction
helper-dependent adenoviral vector
chimeric adenoviral vector
cerebellum
Purkinje cell
On-line přístup:http://www.sciencedirect.com/science/article/pii/S2329050124000597
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