Overloaded Adeno-Associated Virus as a Novel Gene Therapeutic Tool for Otoferlin-Related Deafness

Hearing impairment is the most common sensory disorder in humans. So far, rehabilitation of profoundly deaf subjects relies on direct stimulation of the auditory nerve through cochlear implants. However, in some forms of genetic hearing impairment, the organ of Corti is structurally intact and thera...

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Bibliographic Details
Published in:Frontiers in molecular neuroscience 2021-01, Vol.13, p.600051-600051
Main Authors: Rankovic, Vladan, Vogl, Christian, Dörje, Nele M, Bahader, Iman, Duque-Afonso, Carlos J, Thirumalai, Anupriya, Weber, Thomas, Kusch, Kathrin, Strenzke, Nicola, Moser, Tobias
Format: Article
Language:English
Subjects:
AAV (adeno-associated virus)
Auditory nerve
Auditory Neuroscience
Brain stem
Cloning
Cochlea
Deafness
deafness/hearing loss
Enzymes
Gene therapy
gene therapy and therapeutic delivery
Hearing loss
Immunofluorescence
Neuroscience
Organ of Corti
otoferlin
Polymerase chain reaction
preclinical
Rehabilitation
Vectors (Biology)
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Summary:Hearing impairment is the most common sensory disorder in humans. So far, rehabilitation of profoundly deaf subjects relies on direct stimulation of the auditory nerve through cochlear implants. However, in some forms of genetic hearing impairment, the organ of Corti is structurally intact and therapeutic replacement of the mutated gene could potentially restore near natural hearing. In the case of defects of the otoferlin gene ( ), such gene therapy is hindered by the size of the coding sequence (~6 kb) exceeding the cargo capacity (
ISSN:1662-5099
1662-5099
DOI:10.3389/fnmol.2020.600051