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Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases

Authors :
Leyao Li
Lakshmy Vasan
Bryan Kartono
Kevan Clifford
Ahmadreza Attarpour
Raghav Sharma
Matthew Mandrozos
Ain Kim
Wenda Zhao
Ari Belotserkovsky
Claire Verkuyl
Gerold Schmitt-Ulms
Source :
Biomedicines, Vol 11, Iss 10, p 2725 (2023)
Publication Year :
2023
Publisher :
MDPI AG, 2023.

Abstract

Recombinant adeno-associated virus (rAAV) vectors are gene therapy delivery tools that offer a promising platform for the treatment of neurodegenerative diseases. Keeping up with developments in this fast-moving area of research is a challenge. This review was thus written with the intention to introduce this field of study to those who are new to it and direct others who are struggling to stay abreast of the literature towards notable recent studies. In ten sections, we briefly highlight early milestones within this field and its first clinical success stories. We showcase current clinical trials, which focus on gene replacement, gene augmentation, or gene suppression strategies. Next, we discuss ongoing efforts to improve the tropism of rAAV vectors for brain applications and introduce pre-clinical research directed toward harnessing rAAV vectors for gene editing applications. Subsequently, we present common genetic elements coded by the single-stranded DNA of rAAV vectors, their so-called payloads. Our focus is on recent advances that are bound to increase treatment efficacies. As needed, we included studies outside the neurodegenerative disease field that showcased improved pre-clinical designs of all-in-one rAAV vectors for gene editing applications. Finally, we discuss risks associated with off-target effects and inadvertent immunogenicity that these technologies harbor as well as the mitigation strategies available to date to make their application safer.

Details

Language :
English
ISSN :
22279059
Volume :
11
Issue :
10
Database :
Directory of Open Access Journals
Journal :
Biomedicines
Publication Type :
Academic Journal
Accession number :
edsdoj.1cdbe5a687841bbbecf59371bc06b5f
Document Type :
article
Full Text :
https://doi.org/10.3390/biomedicines11102725