Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease

Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease

Abstract Deafness affects 5% of the world's population, yet there is a lack of treatments to prevent hearing loss due to genetic causes. Norrie disease is a recessive X‐linked disorder, caused by NDP gene mutation. It manifests as blindness at birth and progressive sensorineural hearing loss, l...

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Bibliographic Details
Main Authors: Valda Pauzuolyte, Aara Patel, James R Wawrzynski, Neil J Ingham, Yeh Chwan Leong, Rajvinder Karda, Maria Bitner‐Glindzicz, Wolfgang Berger, Simon N Waddington, Karen P Steel, Jane C Sowden
Format: Article
Language:English
Published: Springer Nature 2023-10-01
Series:EMBO Molecular Medicine
Subjects:
AAV9 gene therapy
cochlea
Ndp
retina
vascular
Online Access:https://doi.org/10.15252/emmm.202317393

Internet

https://doi.org/10.15252/emmm.202317393

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