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

Björklund, Tomas LU and Davidsson, Marcus LU (2021) In Journal of Parkinson's Disease 11(s2). p.209-217
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... (More)

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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Please use this url to cite or link to this publication:
author
and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
clinical trial, dependovirus, gene editing, Genetic therapy, neuroprotection, Parkinson's disease, rejuvenation
in
Journal of Parkinson's Disease
volume
11
issue
s2
pages
209 - 217
publisher
IOS Press
external identifiers
  • pmid:34366370
  • scopus:85116029721
ISSN
1877-7171
DOI
10.3233/jpd-212674
language
English
LU publication?
yes
additional info
Publisher Copyright: © 2021 - The authors. Published by IOS Press.
id
f0357f7f-6cea-42d8-a5f7-c3f73f28e237
date added to LUP
2021-10-22 16:01:17
date last changed
2025-01-13 16:00:39
@article{f0357f7f-6cea-42d8-a5f7-c3f73f28e237,
  abstract     = {{<p>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.</p>}},
  author       = {{Björklund, Tomas and Davidsson, Marcus}},
  issn         = {{1877-7171}},
  keywords     = {{clinical trial; dependovirus; gene editing; Genetic therapy; neuroprotection; Parkinson's disease; rejuvenation}},
  language     = {{eng}},
  number       = {{s2}},
  pages        = {{209--217}},
  publisher    = {{IOS Press}},
  series       = {{Journal of Parkinson's Disease}},
  title        = {{Next-Generation Gene Therapy for Parkinson's Disease Using Engineered Viral Vectors}},
  url          = {{http://dx.doi.org/10.3233/jpd-212674}},
  doi          = {{10.3233/jpd-212674}},
  volume       = {{11}},
  year         = {{2021}},
}