Next-Generation Gene Therapy for Parkinson's Disease Using Engineered Viral Vectors

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Sammanfattning

Recent technological and conceptual advances have resulted in a plethora of exciting novel engineered adeno associated viral (AAV) vector variants. They all have unique characteristics and abilities. This review summarizes the development and their potential in treating Parkinson's disease (PD). Clinical trials in PD have shown over the last decade that AAV is a safe and suitable vector for gene therapy but that it also is a vehicle that can benefit significantly from improvement in specificity and potency. This review provides a concise collection of the state-of-the-art for synthetic capsids and their utility in PD. We also summarize what therapeutical strategies may become feasible with novel engineered vectors, including genome editing and neuronal rejuvenation.

Originalspråkengelska
Sidor (från-till)S209-S217
TidskriftJournal of Parkinson's Disease
Volym11
Nummers2
DOI
StatusPublished - 2021

Bibliografisk information

Publisher Copyright:
© 2021 - The authors. Published by IOS Press.

Ämnesklassifikation (UKÄ)

  • Neurovetenskaper

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