Advances in genome editing for genetic hearing loss

Ning Ding; Sangsin Lee; Matan Lieber-Kotz; Jie Yang; Xue Gao

doi:10.1016/j.addr.2020.05.001

Advances in genome editing for genetic hearing loss

Adv Drug Deliv Rev. 2021 Jan:168:118-133. doi: 10.1016/j.addr.2020.05.001. Epub 2020 May 7.

Authors

Ning Ding¹, Sangsin Lee², Matan Lieber-Kotz³, Jie Yang⁴, Xue Gao⁵

Affiliations

¹ Department of Chemical and Biomolecular Engineering, Rice University, Houston, Texas 77005, USA; School of Food Science and Technology, Jiangnan University, Wuxi, Jiangsu 214122, China. Electronic address: novyanna_ding@126.com.
² Department of Bioengineering, Rice University, Houston, Texas 77030, USA. Electronic address: sl120@rice.edu.
³ Department of Chemical and Biomolecular Engineering, Rice University, Houston, Texas 77005, USA.
⁴ Department of Chemical and Biomolecular Engineering, Rice University, Houston, Texas 77005, USA; Department of Biochemistry and Molecular Biology, School of Basic Medical Sciences, Tianjin Medical University, Tianjin 300072, China.
⁵ Department of Chemical and Biomolecular Engineering, Rice University, Houston, Texas 77005, USA; Department of Bioengineering, Rice University, Houston, Texas 77030, USA. Electronic address: xue.gao@rice.edu.

Abstract

According to the World Health Organization, hearing loss affects over 466 million people worldwide and is the most common human sensory impairment. It is estimated that genetic factors contribute to the causation of approximately 50% of congenital hearing loss. Yet, curative approaches to reversing or preventing genetic hearing impairment are still limited. The clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) systems enable programmable and targeted gene editing in highly versatile manners and offer new gene therapy strategies for genetic hearing loss. Here, we summarize the most common deafness-associated genes, illustrate recent strategies undertaken by using CRISPR-Cas9 systems for targeted gene editing and further compare the CRISPR strategies to non-CRISPR gene therapies. We also examine the merits of different vehicles and delivery forms of genome editing agents. Lastly, we describe the development of animal models that could facilitate the eventual clinical applications of the CRISPR technology to the treatment of genetic hearing diseases.

Keywords: Base editor; CRISPR-Cas9; Gene delivery; Gene therapy; Genome editing; Hearing loss.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Clustered Regularly Interspaced Short Palindromic Repeats*
Gene Editing / methods*
Genetic Therapy / methods*
Hearing Loss / genetics*
Hearing Loss / therapy*
Polymorphism, Single Nucleotide

Abstract

Publication types

MeSH terms

Grants and funding