Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation

W Zhu, W Du, AP Rameshbabu… - Science translational …, 2024 - science.org
Mutations in microRNA-96 (MIR96) cause autosomal dominant deafness-50 (DFNA50), a
form of delayed-onset hearing loss. Genome editing has shown efficacy in hearing recovery …

[HTML][HTML] CRISPR-mediated RNA base editing: a promising strategy to rescue deafness

W Liu, M Wu, G Shen - Signal Transduction and Targeted Therapy, 2023 - nature.com
A recent study published in Science Translational Medicine by Xiao et al. reported the use of
mini dCas13X-based adenosine base editor (mxABE) to correct a mutant transcript and …

Rescue of autosomal dominant hearing loss by in vivo delivery of mini dCas13X-derived RNA base editor

Q Xiao, Z Xu, Y Xue, C Xu, L Han, Y Liu… - Science Translational …, 2022 - science.org
Programmable RNA editing tools enable the reversible correction of mutant transcripts,
reducing the potential risk associated with permanent genetic changes associated with the …

[HTML][HTML] Preventing autosomal-dominant hearing loss in Bth mice with CRISPR/CasRx-based RNA editing

Z Zheng, G Li, C Cui, F Wang, X Wang, Z Xu… - … and Targeted Therapy, 2022 - nature.com
Abstract CRISPR/RfxCas13d (CasRx) editing system can specifically and precisely cleave
single-strand RNAs, which is a promising treatment for various disorders by downregulation …

[HTML][HTML] Prevention of acquired sensorineural hearing loss in mice by in vivo Htra2 gene editing

X Gu, D Wang, Z Xu, J Wang, L Guo, R Chai, G Li… - Genome Biology, 2021 - Springer
Background Aging, noise, infection, and ototoxic drugs are the major causes of human
acquired sensorineural hearing loss, but treatment options are limited. CRISPR/Cas9 …

[HTML][HTML] In vivo outer hair cell gene editing ameliorates progressive hearing loss in dominant-negative Kcnq4 murine model

B Noh, JH Rim, R Gopalappa, H Lin, KM Kim… - Theranostics, 2022 - ncbi.nlm.nih.gov
Outer hair cell (OHC) degeneration is a major cause of progressive hearing loss and
presbycusis. Despite the high prevalence of these disorders, targeted therapy is currently …

CRISPR/Cas9: targeted genome editing for the treatment of hereditary hearing loss

R Farooq, K Hussain, M Tariq, A Farooq… - Journal of Applied …, 2020 - Springer
Hereditary hearing loss (HHL) is a neurosensory disorder that affects every 1/500 newborns
worldwide and nearly 1/3 people over the age of 65. Congenital deafness is inherited as …

[HTML][HTML] Gene editing in a Myo6 semi-dominant mouse model rescues auditory function

Y Xue, X Hu, D Wang, D Li, Y Li, F Wang, M Huang… - Molecular Therapy, 2022 - cell.com
Myosin VI (MYO6) is an unconventional myosin that is vital for auditory and vestibular
function. Pathogenic variants in the human MYO6 gene cause autosomal-dominant or …

The application of genome editing in studying hearing loss

B Zou, R Mittal, M Grati, Z Lu, Y Shu, Y Tao, Y Feng… - Hearing research, 2015 - Elsevier
Targeted genome editing mediated by clustered, regularly interspaced, short palindromic
repeat (CRISPR)/CRISPR-associated nuclease 9 (Cas9) technology has emerged as one of …

Characterization of promoters for adeno-associated virus mediated efficient Cas9 activation in adult Cas9 knock-in murine cochleae

X Zhao, C Jin, T Dong, Z Sun, X Zheng, B Feng… - Hearing Research, 2020 - Elsevier
CRISPR/Cas9 gene editing enables the treatment of hearing loss in congenitally deaf
neonatal mice via both viral and non-viral delivery. While adeno-associated virus (AAV) …