Breaking genetic shackles: The advance of base editing in genetic disorder treatment
F Xu, C Zheng, W Xu, S Zhang, S Liu… - Frontiers in …, 2024 - frontiersin.org
The rapid evolution of gene editing technology has markedly improved the outlook for
treating genetic diseases. Base editing, recognized as an exceptionally precise genetic …
treating genetic diseases. Base editing, recognized as an exceptionally precise genetic …
In vivo CRISPR base editing for treatment of Huntington′ s disease
Huntington's disease (HD) is an inherited and ultimately fatal neurodegenerative disorder
caused by an expanded polyglutamine-encoding CAG repeat within exon 1 of the huntingtin …
caused by an expanded polyglutamine-encoding CAG repeat within exon 1 of the huntingtin …
SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping
Exon skipping technologies enable exclusion of targeted exons from mature mRNA
transcripts, which has broad applications in molecular biology, medicine, and biotechnology …
transcripts, which has broad applications in molecular biology, medicine, and biotechnology …