Literature DB >> 32387678

Advances in genome editing for genetic hearing loss.

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

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.
Copyright © 2020 Elsevier B.V. All rights reserved.

Entities:  

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

Mesh:

Year:  2020        PMID: 32387678      PMCID: PMC7647959          DOI: 10.1016/j.addr.2020.05.001

Source DB:  PubMed          Journal:  Adv Drug Deliv Rev        ISSN: 0169-409X            Impact factor:   15.470


  165 in total

Review 1.  Comparative anatomy of the cochlea and auditory nerve in mammals.

Authors:  J B Nadol
Journal:  Hear Res       Date:  1988-08       Impact factor: 3.208

2.  Utilizing ethnic-specific differences in minor allele frequency to recategorize reported pathogenic deafness variants.

Authors:  A Eliot Shearer; Robert W Eppsteiner; Kevin T Booth; Sean S Ephraim; José Gurrola; Allen Simpson; E Ann Black-Ziegelbein; Swati Joshi; Harini Ravi; Angelica C Giuffre; Scott Happe; Michael S Hildebrand; Hela Azaiez; Yildirim A Bayazit; Mehmet Emin Erdal; Jose A Lopez-Escamez; Irene Gazquez; Marta L Tamayo; Nancy Y Gelvez; Greizy Lopez Leal; Chaim Jalas; Josef Ekstein; Tao Yang; Shin-ichi Usami; Kimia Kahrizi; Niloofar Bazazzadegan; Hossein Najmabadi; Todd E Scheetz; Terry A Braun; Thomas L Casavant; Emily M LeProust; Richard J H Smith
Journal:  Am J Hum Genet       Date:  2014-09-25       Impact factor: 11.025

3.  Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9.

Authors:  Dominik Paquet; Dylan Kwart; Antonia Chen; Andrew Sproul; Samson Jacob; Shaun Teo; Kimberly Moore Olsen; Andrew Gregg; Scott Noggle; Marc Tessier-Lavigne
Journal:  Nature       Date:  2016-04-27       Impact factor: 49.962

4.  Overlapping expression of anion exchangers in the cochlea of a non-human primate suggests functional compensation.

Authors:  Makoto Hosoya; Masato Fujioka; Reona Kobayashi; Hideyuki Okano; Kaoru Ogawa
Journal:  Neurosci Res       Date:  2016-04-19       Impact factor: 3.304

5.  Gene transfer into guinea pig cochlea using adeno-associated virus vectors.

Authors:  Masaya Konishi; Kohei Kawamoto; Masahiko Izumikawa; Hiromichi Kuriyama; Toshio Yamashita
Journal:  J Gene Med       Date:  2008-06       Impact factor: 4.565

6.  Gene Therapy Restores Hair Cell Stereocilia Morphology in Inner Ears of Deaf Whirler Mice.

Authors:  Wade W Chien; Kevin Isgrig; Soumen Roy; Inna A Belyantseva; Meghan C Drummond; Lindsey A May; Tracy S Fitzgerald; Thomas B Friedman; Lisa L Cunningham
Journal:  Mol Ther       Date:  2015-08-26       Impact factor: 11.454

7.  Polyethylenimine-mediated cochlear gene transfer in guinea pigs.

Authors:  Brian Tiong Gee Tan; Kok Heng Foong; Myranda Mui Gek Lee; Runsheng Ruan
Journal:  Arch Otolaryngol Head Neck Surg       Date:  2008-08

Review 8.  Antisense oligonucleotides: the next frontier for treatment of neurological disorders.

Authors:  Carlo Rinaldi; Matthew J A Wood
Journal:  Nat Rev Neurol       Date:  2017-12-01       Impact factor: 42.937

Review 9.  The DFNB1 subtype of autosomal recessive non-syndromic hearing impairment.

Authors:  Francisco J del Castillo; Ignacio del Castillo
Journal:  Front Biosci (Landmark Ed)       Date:  2011-06-01

10.  Adenovirus Vectors Target Several Cell Subtypes of Mammalian Inner Ear In Vivo.

Authors:  Yilai Shu; Yong Tao; Wenyan Li; Jun Shen; Zhengmin Wang; Zheng-Yi Chen
Journal:  Neural Plast       Date:  2016-12-28       Impact factor: 3.599

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  6 in total

Review 1.  Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics.

Authors:  Wenyi Liu; Luoxi Li; Jianxin Jiang; Min Wu; Ping Lin
Journal:  Precis Clin Med       Date:  2021-07-10

Review 2.  Conversations in Cochlear Implantation: The Inner Ear Therapy of Today.

Authors:  Grant Rauterkus; Anne K Maxwell; Jacob B Kahane; Jennifer J Lentz; Moises A Arriaga
Journal:  Biomolecules       Date:  2022-04-29

Review 3.  How Transmembrane Inner Ear (TMIE) plays role in the auditory system: A mystery to us.

Authors:  Mohammad Farhadi; Ehsan Razmara; Maryam Balali; Yeganeh Hajabbas Farshchi; Masoumeh Falah
Journal:  J Cell Mol Med       Date:  2021-05-13       Impact factor: 5.310

4.  In vivo outer hair cell gene editing ameliorates progressive hearing loss in dominant-negative Kcnq4 murine model.

Authors:  Byunghwa Noh; John Hoon Rim; Ramu Gopalappa; Haiyue Lin; Kyu Min Kim; Min Jin Kang; Heon Yung Gee; Jae Young Choi; Hyongbum Henry Kim; Jinsei Jung
Journal:  Theranostics       Date:  2022-02-28       Impact factor: 11.556

5.  Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss.

Authors:  Chong Cui; Daqi Wang; Bowei Huang; Fang Wang; Yuxin Chen; Jun Lv; Luping Zhang; Lei Han; Dong Liu; Zheng-Yi Chen; Geng-Lin Li; Huawei Li; Yilai Shu
Journal:  Mol Ther Nucleic Acids       Date:  2022-07-20       Impact factor: 10.183

6.  Analysis of TMIE gene mutations including the first large deletion of exon 1 with autosomal recessive non-syndromic deafness.

Authors:  Sima Rayat; Mohammad Farhadi; Hessamaldin Emamdjomeh; Saeid Morovvati; Masoumeh Falah
Journal:  BMC Med Genomics       Date:  2022-06-16       Impact factor: 3.622

  6 in total

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