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Preclinical studies in mice and non-human primates with a gene therapy designed to deliver GJB2 gene expression in specific cells of the ear open the possibility to treat the most frequent type of hereditary hearing loss in humans
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
Cell-specific delivery of GJB2 restores auditory function in mouse models of DFNB1 deafness and mediates appropriate expression in NHP cochlea
Mutations in the GJB2 gene cause the most common form of human hereditary hearing loss, known as DFNB1. GJB2 is expressed in two cell groups of the cochlea—epithelial cells of the organ of Corti and f...
www.biorxiv.org
January 8, 2025 at 8:14 AM
Preclinical studies in mice and non-human primates with a gene therapy designed to deliver GJB2 gene expression in specific cells of the ear open the possibility to treat the most frequent type of hereditary hearing loss in humans
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
Estudios preclínicos realizados en ratones y en primates con una terapia génica diseñada para reponer la expresión del gen GJB2 en células específicas del oído abren la puerta a la posibilidad de tratar la sordera hereditaria más frecuente en humanos.
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
Cell-specific delivery of GJB2 restores auditory function in mouse models of DFNB1 deafness and mediates appropriate expression in NHP cochlea
Mutations in the GJB2 gene cause the most common form of human hereditary hearing loss, known as DFNB1. GJB2 is expressed in two cell groups of the cochlea—epithelial cells of the organ of Corti and f...
www.biorxiv.org
January 8, 2025 at 8:12 AM
Estudios preclínicos realizados en ratones y en primates con una terapia génica diseñada para reponer la expresión del gen GJB2 en células específicas del oído abren la puerta a la posibilidad de tratar la sordera hereditaria más frecuente en humanos.
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
Thousands of new potential miniproteins might expand the number of functional human genes and help to explain and treat undiagnosed genetic diseases.
‘Dark proteome’ survey reveals thousands of new human genes
Database confirms that overlooked segments of the genome code for a multitude of tiny proteins
www.science.org
November 29, 2024 at 8:06 AM
Thousands of new potential miniproteins might expand the number of functional human genes and help to explain and treat undiagnosed genetic diseases.