Editing the Central Nervous System Through CRISPR/Cas9 Systems

The translational gap to treatments based on gene therapy has been reduced in recent years because of improvements in gene editing tools, such as the CRISPR/Cas9 system and its variations. This has allowed the development of more precise therapies for neurodegenerative diseases, where access is priv...

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Bibliographic Details
Published in:Frontiers in molecular neuroscience 2019-05, Vol.12, p.110-110
Main Authors: Cota-Coronado, Agustin, Díaz-Martínez, Néstor Fabián, Padilla-Camberos, Eduardo, Díaz-Martínez, N Emmanuel
Format: Article
Language:English
Subjects:
adenovirus-associated virus
Alzheimer's disease
Blood-brain barrier
Brain research
Central nervous system
CRISPR
CRISPR/Cas9
d-Cas9
Deoxyribonucleic acid
Design
Disease
DNA
Efficiency
Enzymes
Gene therapy
Genomes
Mutation
Nanoparticles
Neurodegeneration
Neurodegenerative diseases
Neuroscience
“Trojan horse” peptides
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Summary:The translational gap to treatments based on gene therapy has been reduced in recent years because of improvements in gene editing tools, such as the CRISPR/Cas9 system and its variations. This has allowed the development of more precise therapies for neurodegenerative diseases, where access is privileged. As a result, engineering of complexes that can access the central nervous system (CNS) with the least potential inconvenience is fundamental. In this review article, we describe current alternatives to generate systems based on CRISPR/Cas9 that can cross the blood-brain barrier (BBB) and may be used further clinically to improve treatment for neurodegeneration in Parkinson's and Alzheimer's disease (AD).
ISSN:1662-5099
1662-5099
DOI:10.3389/fnmol.2019.00110