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

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, leading to...

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Detalles Bibliográficos
Autores principales: Pauzuolyte, Valda, Patel, Aara, Wawrzynski, James R, Ingham, Neil J, Leong, Yeh Chwan, Karda, Rajvinder, Bitner‐Glindzicz, Maria, Berger, Wolfgang, Waddington, Simon N, Steel, Karen P, Sowden, Jane C
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10565640/
https://www.ncbi.nlm.nih.gov/pubmed/37642150
http://dx.doi.org/10.15252/emmm.202317393

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10565640/
https://www.ncbi.nlm.nih.gov/pubmed/37642150
http://dx.doi.org/10.15252/emmm.202317393

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