Stem Cells and Gene Therapy in Progressive Hearing Loss: the State of the Art

Autor: Karl R. Koehler, Aida Nourbakhsh, Aziz El-Amraoui, Xue Zhong Liu, Eric Nisenbaum, Brett Colbert, Derek M. Dykxhoorn, Zheng-Yi Chen
Přispěvatelé: HAL-SU, Gestionnaire, ECLAIRER LA SURDITÉ : UNE APPROCHE HOLISTIQUE DU SYNDROME D'USHER - - LIGHT4DEAF2015 - ANR-15-RHUS-0001 - RHUS - VALID, Surdité d'apparition tardive et progressive: de la physiopathologie à la thérapie - - HearInNoise2017 - ANR-17-CE16-0017 - AAPG2017 - VALID, University of Miami Leonard M. Miller School of Medicine (UMMSM), Déficits Sensoriels Progressifs, Pathophysiologie et Thérapie / Progressive Sensory Disorders, PathoPhysiology and Therapy, Institut Pasteur [Paris] (IP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), Harvard Medical School [Boston] (HMS), Dr. Liu’s lab is supported by NIH grants of R01DC005575, R01DC012115, and R01DC107624. Drs. Aida Nourbakhsh and Eric Nisenbaum are supported by T32 DC015995. Dr. El-Amraoui’s lab is supported by French National Research Agency (ANR) as part of the second 'Investissements d’Avenir' programme (light4deaf, ANR-15-RHUS-0001), ANR-HearInNoise-(ANR-17-CE16-0017), and LHW-Stiftung., ANR-15-RHUS-0001,LIGHT4DEAF,ECLAIRER LA SURDITÉ : UNE APPROCHE HOLISTIQUE DU SYNDROME D'USHER(2015), ANR-17-CE16-0017,HearInNoise,Surdité d'apparition tardive et progressive: de la physiopathologie à la thérapie(2017), Institut Pasteur [Paris]-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Zdroj: Journal of the Association for Research in Otolaryngology
Journal of the Association for Research in Otolaryngology, 2021, 22 (2), pp.95-105. ⟨10.1007/s10162-020-00781-0⟩
JARO: Journal of the Association for Research in Otolaryngology
Journal of the Association for Research in Otolaryngology, Springer Verlag, 2021, 22 (2), pp.95-105. ⟨10.1007/s10162-020-00781-0⟩
ISSN: 1525-3961
1438-7573
DOI: 10.1007/s10162-020-00781-0⟩
Popis: Progressive non-syndromic sensorineural hearing loss (PNSHL) is the most common cause of sensory impairment, affecting more than a third of individuals over the age of 65. PNSHL includes noise-induced hearing loss (NIHL) and inherited forms of deafness, among which is delayed-onset autosomal dominant hearing loss (AD PNSHL). PNSHL is a prime candidate for genetic therapies due to the fact that PNSHL has been studied extensively, and there is a potentially wide window between identification of the disorder and the onset of hearing loss. Several gene therapy strategies exist that show potential for targeting PNSHL, including viral and non-viral approaches, and gene editing versus gene-modulating approaches. To fully explore the potential of these therapy strategies, a faithful in vitro model of the human inner ear is needed. Such models may come from induced pluripotent stem cells (iPSCs). The development of new treatment modalities by combining iPSC modeling with novel and innovative gene therapy approaches will pave the way for future applications leading to improved quality of life for many affected individuals and their families.
Databáze: OpenAIRE
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