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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2021
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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. |
format | Online Article Text |
id | pubmed-8543274 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | IOS Press |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT bjorklundtomas nextgenerationgenetherapyforparkinsonsdiseaseusingengineeredviralvectors AT davidssonmarcus nextgenerationgenetherapyforparkinsonsdiseaseusingengineeredviralvectors |