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

ABSTRACT: 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.

SUBMITTER: Bjorklund T

PROVIDER: S-EPMC8543274 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature

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

Björklund Tomas T Davidsson Marcus M

Journal of Parkinson's disease 20210101 s2

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 ...[more]

PMID: 34366370

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