Removal of a partial genomic duplication restores synaptic transmission and behavior in the MyosinVA mutant mouse Flailer.

Fernando Jose BustosSwarna PandianHenny HaensgenJian-Ping ZhaoHaley StroufMatthias HeidenreichLukasz SwiechBenjamin E DevermanViviana GradinaruFeng ZhangMartha Constantine-Paton

Published in: BMC biology (2023)

Our results demonstrate the potential of DN-CRISPR to efficiently remove larger genomic duplications, working as a new gene therapy approach for treating neurodegenerative diseases.

Keyphrases

gene therapy
copy number
genome wide
crispr cas
genome editing
dna methylation
wild type
gene expression
risk assessment