Application of CRISPR/Cas system in iPSC-based disease model of hereditary deafness 9

CRISPR/Cas9; Deafness; Disease modeling; Gene editing; Gene therapy; Hearing loss; Human embryonic stem cell; Induced pluripotent stem cell; Inner ear hair cell; Organoid; Otic progenitors; Sensorineural

Abstract :

[en] Sensorineural hearing loss (SNHL) is the most prevalent sensory disorders affecting w 6% of the world population with partial or complete hearing impairment. The molecular etiologies of SNHL could be inherited genetic or acquired environmental factors. The broadening spectrum of genetic causes of SNHL requires a better understanding of the disease pathogenesis and the development of therapeutic targets. To achieve this, we need an experimental humanoid disease model that is fast and easy to perform genetic manipulation and downstream. In this chapter, we discuss how two revolutionary techniques CRISPR/Cas-based gene editing and human-induced pluripotent cells (hiPSCs) could be implemented and reshape the experimental disease model of hearing loss with a vision toward the development of reliable and sustainable therapeutic approaches.

Research Center/Unit :

GIGA Stem Cells-Developmental Neurobiology - ULiège

Disciplines :

Otolaryngology
Biochemistry, biophysics & molecular biology

Author, co-author :

Giri, Subhajit ; Université de Liège - ULiège > Département des sciences cliniques > Neurologie

Malgrange, Brigitte ; Université de Liège - ULiège > GIGA > GIGA Neurosciences

Language :

English

Title :

Application of CRISPR/Cas system in iPSC-based disease model of hereditary deafness 9

Publication date :

2022

Main work title :

iPSCs - State of the Science

Author, co-author :

Birbrair, Alexander

Publisher :

academic press

ISBN/EAN :

978-0-323-85767-3

Collection name :

Volume 16 in Advances in Stem Cell Biology

Peer reviewed :

Peer reviewed

Available on ORBi :

since 15 March 2022

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71 (2 by ULiège)

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133 (2 by ULiège)

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