Back to Search
Start Over
AAV-ie enables safe and efficient gene transfer to inner ear cells
- Source :
- Nature Communications, Nature Communications, Vol 10, Iss 1, Pp 1-10 (2019)
- Publication Year :
- 2019
-
Abstract
- Hearing loss is the most common sensory disorder. While gene therapy has emerged as a promising treatment of inherited diseases like hearing loss, it is dependent on the identification of gene delivery vectors. Adeno-associated virus (AAV) vector-mediated gene therapy has been approved in the US for treating a rare inherited eye disease but no safe and efficient vectors have been identified that can target the diverse types of inner ear cells. Here, we identify an AAV variant, AAV-inner ear (AAV-ie), for gene delivery in mouse inner ear. Our results show that AAV-ie transduces the cochlear supporting cells (SCs) with high efficiency, representing a vast improvement over conventional AAV serotypes. Furthermore, after AAV-ie-mediated transfer of the Atoh1 gene, we find that many SCs trans-differentiated into new HCs. Our results suggest that AAV-ie is a useful tool for the cochlear gene therapy and for investigating the mechanism of HC regeneration.<br />There are currently few AAV vectors that can effectively target the diverse cell types of the inner ear. Here the authors design AAV-ie for gene delivery to the mouse cochlea, targeting cochlear supporting cells.
- Subjects :
- 0301 basic medicine
Male
Hearing loss
Genetic enhancement
Science
viruses
Genetic Vectors
General Physics and Astronomy
ATOH1 Gene
02 engineering and technology
Gene delivery
Biology
General Biochemistry, Genetics and Molecular Biology
Virus
Article
03 medical and health sciences
Mice
medicine
otorhinolaryngologic diseases
Basic Helix-Loop-Helix Transcription Factors
Animals
Inner ear
lcsh:Science
Hearing Loss
Cochlea
Cells, Cultured
Multidisciplinary
Hair Cells, Auditory, Inner
Regeneration (biology)
Gene Transfer Techniques
General Chemistry
Genetic Therapy
Dependovirus
021001 nanoscience & nanotechnology
Cell biology
Mice, Inbred C57BL
030104 developmental biology
medicine.anatomical_structure
lcsh:Q
Female
sense organs
medicine.symptom
0210 nano-technology
Subjects
Details
- ISSN :
- 20411723
- Volume :
- 10
- Issue :
- 1
- Database :
- OpenAIRE
- Journal :
- Nature communications
- Accession number :
- edsair.doi.dedup.....7ebd7cf3f6ce4c0efb4a2944e48ad62e