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

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). Cli...

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Detalles Bibliográficos
Autores principales: Björklund, Tomas, Davidsson, Marcus
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8543274/
https://www.ncbi.nlm.nih.gov/pubmed/34366370
http://dx.doi.org/10.3233/JPD-212674
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author Björklund, Tomas
Davidsson, Marcus
author_facet Björklund, Tomas
Davidsson, Marcus
author_sort Björklund, Tomas
collection PubMed
description 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.
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spelling pubmed-85432742021-11-10 Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors Björklund, Tomas Davidsson, Marcus J Parkinsons Dis Review 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. IOS Press 2021-09-22 /pmc/articles/PMC8543274/ /pubmed/34366370 http://dx.doi.org/10.3233/JPD-212674 Text en © 2021 – The authors. Published by IOS Press https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Björklund, Tomas
Davidsson, Marcus
Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title_full Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title_fullStr Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title_full_unstemmed Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title_short Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
title_sort next-generation gene therapy for parkinson’s disease using engineered viral vectors
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8543274/
https://www.ncbi.nlm.nih.gov/pubmed/34366370
http://dx.doi.org/10.3233/JPD-212674
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