Back to search

Article

Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in <i>USH2A</i> and comparison of methods

2024-10-03

Abstract excerpt

Base editing shows promise for the correction of human mutations at a higher efficiency than other repair methods and is especially attractive for mutations in large genes that are not amenable to gene augmentation therapy. Here, we demonstrate a comprehensive workflow for in vitro screening of potential therapeutic base editing targets for the USH2A gene and empirically validate the efficiency of adenine and cyt...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work
89d50b4f-bf58-5953-8eb9-6ce27fbdc047
DOI
10.1101/2024.10.02.615422
Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest
Interactive article relationship graphSelect a related publication card to move it into the centre and load its closest explainable connections. Solid lines are source-backed structured connections. Dashed lines are semantic discovery signals and are not scientific evidence.
Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in <i>USH2A</i> and comparison of methodsDOI 10.1101/2024.10.02.615422
Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods.Semantic proximity
Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degenerationSemantic proximity
Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseasesSemantic proximity
Breaking genetic shackles: The advance of base editing in genetic disorder treatmentSemantic proximity
PAM-flexible adenine base editing rescues hearing loss in a humanized <i>MPZL2</i> mouse model harboring an East Asian founder mutationSemantic proximity
Precision genome editing using cytosine and adenine base editors in mammalian cells.Semantic proximity
Improved specificity and efficiency of <i>in vivo</i> adenine base editing therapies with hybrid guide RNAsSemantic proximity
Compact adenine base editors to enable therapeutic rescue of Duchenne muscular dystrophySemantic proximity
PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutationSemantic proximity
An engineered U7 small nuclear RNA scaffold greatly increases ADAR-mediated programmable RNA base editingSemantic proximity
Template-independent genome editing and repairing correct frameshift disease <i>in vivo</i>Semantic proximity
Precise <i>in vivo</i> functional analysis of DNA variants with base editing using ACEofBASEs target predictionSemantic proximity
Select a neighboring publication to make it the new centre.