Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Apr 2025

Tachida Yuki, Manian Kannan V, Butcher Rossano, Levy Jonathan M, Pendse Nachiket, Hennessey Erin, Liu David R, Pierce Eric A, Liu Qin, Comander Jason

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...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CRISPR-Cas Systems
Share to Dependovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Disease Models, Animal

Share to Extracellular Matrix Proteins

Share to Gene Editing

Share to Genetic Therapy

Share to High-Throughput Nucleotide Sequencing

Share to Usher Syndromes

Source

Open in PubMed Open full text

Identifiers

PMID: 39881543
DOI: 10.1016/j.ymthe.2025.01.042
PMCID: PMC11997516

Genes

USH2A

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods.PMID 39881543Read ↗

Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in <i>USH2A</i> and comparison of methodsSemantic proximity Read ↗

Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degenerationSemantic proximity Read ↗

Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseasesSemantic proximity Read ↗

Breaking genetic shackles: The advance of base editing in genetic disorder treatmentSemantic proximity Read ↗

Precision genome editing using cytosine and adenine base editors in mammalian cells.Semantic proximity Read ↗

[Advances on gene therapy for USH2A exon 13 related inherited retinal dystrophy].Semantic proximity Read ↗

Compact adenine base editors to enable therapeutic rescue of Duchenne muscular dystrophySemantic proximity Read ↗

An engineered U7 small nuclear RNA scaffold greatly increases ADAR-mediated programmable RNA base editingSemantic proximity Read ↗

Adenine base editing is an efficient approach to restore function in FA patient cells without double-stranded DNA breaksSemantic proximity Read ↗

Improved specificity and efficiency of <i>in vivo</i> adenine base editing therapies with hybrid guide RNAsSemantic proximity Read ↗

ABE-ultramax for high-efficiency biallelic adenine base editing in zebrafish.Semantic proximity Read ↗

PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutationSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.