Open AccessNext-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors
Author(s) -
Tomas Björklund,
Marcus Davidsson
Publication year - 2021
Publication title -
journal of parkinson's disease/journal of parkinson's disease (online)
Language(s) - English
Resource type - Journals
eISSN - 1877-718X
pISSN - 1877-7171
DOI - 10.3233/jpd-212674
Subject(s) - genetic enhancement , parkinson's disease , vector (molecular biology) , viral vector , computational biology , disease , clinical trial , adeno associated virus , genome editing , genome , medicine , neuroscience , biology , bioinformatics , gene , genetics , recombinant dna , pathology
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.