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

Hereditary deafness lacks effective biological treatments. Here, the authors develop a base editing therapy that corrects a common MPZL2 deafness gene mutation in a humanized mouse model and restores hearing, highlighting base editing as a potential treatment for hereditary deafness.

Shao Wei Hu
Sohyang Jeong
Yilai Shu
ResearchOpen Access05 Aug 2025
Nature Communications

Volume: 16, P: 1-18
Abasic pivot substitution harnesses target specificity of RNA interference

RNA interference inadvertently represses off-target transcripts. Here, Lee et al.report that substituting nucleotide in position 6 of the seed region of the small interfering RNAs with abasic spacers can significantly decrease miRNA-like off-target repression while preserving on-target activity.

Hye-Sook Lee
Heeyoung Seok
Sung Wook Chi
ResearchOpen Access18 Dec 2015
Nature Communications

Volume: 6, P: 1-14
Position-specific oxidation of miR-1 encodes cardiac hypertrophy

The 8-oxoguanine modification of the microRNA miR-1 results in redirected recognition and silencing of target genes and leads to cardiac hypertrophy in mice.

Heeyoung Seok
Haejeong Lee
Sung Wook Chi
Research05 Aug 2020
Nature

Volume: 584, P: 279-285
Ramifications of POU4F3 variants associated with autosomal dominant hearing loss in various molecular aspects

Sang-Yeon Lee
Min Young Kim
Byung Yoon Choi
ResearchOpen Access03 Aug 2023
Scientific Reports

Volume: 13, P: 1-14
Factors associated with the difference between the incidence and case-fatality ratio of coronavirus disease 2019 by country

Jeehyun Kim
Kwan Hong
Byung Chul Chun
ResearchOpen Access23 Sept 2021
Scientific Reports

Volume: 11, P: 1-11

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

Abasic pivot substitution harnesses target specificity of RNA interference

Position-specific oxidation of miR-1 encodes cardiac hypertrophy

Ramifications of POU4F3 variants associated with autosomal dominant hearing loss in various molecular aspects

Factors associated with the difference between the incidence and case-fatality ratio of coronavirus disease 2019 by country

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

Abasic pivot substitution harnesses target specificity of RNA interference

Position-specific oxidation of miR-1 encodes cardiac hypertrophy

Ramifications of POU4F3 variants associated with autosomal dominant hearing loss in various molecular aspects

Factors associated with the difference between the incidence and case-fatality ratio of coronavirus disease 2019 by country

Search

Quick links