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Gene editing based hearing impairment research and therapeutics
Wen Kang1, Zhuoer Sun1, Xingle Zhao1
1Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiaotong University School of Medicine, China; Ear Institute, Shanghai Jiaotong University School of Medicine, China; Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases, China.
Neuroscience Letters
|June 14, 2019
Summary
Gene editing technologies like CRISPR-Cas9 offer new hope for treating hereditary hearing loss. These advanced tools are being explored for their potential to restore natural sound perception in the inner ear.
Area of Science:
- Genetics
- Otolaryngology
- Molecular Biology
Background:
- Hearing impairment is a global health concern, affecting newborns and the elderly.
- Hereditary factors are a primary cause of congenital deafness and influence susceptibility.
- Gene therapy shows promise in preclinical models for restoring hearing.
Purpose of the Study:
- To review recent advancements in gene editing for inner ear applications.
- To highlight the potential of gene editing technologies in addressing hearing impairment.
Main Methods:
- Focus on CRISPR-Cas9 and base editors (BEs) as key gene editing tools.
- Discuss their application in studying inner ear genetics.
- Explore their therapeutic potential for hearing loss.
Main Results:
- Gene editing technologies facilitate genetic studies in the inner ear.
- These technologies have shown success in rescuing deafness in hereditary murine models.
- Recent applications demonstrate progress in developing therapeutic strategies.
Conclusions:
- Gene editing presents a promising frontier for treating hearing impairment.
- CRISPR-Cas9 and base editors are pivotal in advancing inner ear research and therapy.
- Further development holds potential for restoring natural sound perception.
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