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NOVEL GENE THERAPY TECHNIQUE TO TREAT HEREDITARY DEAFNESS
- Date Issued:
- 2020
- Abstract/Description:
- Autosomal recessive deafness-9 (DFNB9), caused by mutations in the otoferlin gene (OTOF), is the most common form of hereditary deafness, accounting for 2-8% of all cases. Here, I review recent research on using dual adeno associated virus (AAV) mediated gene therapy to treat DFNB9 in a mouse model system. Dual AAV gene therapy repairs these mutations by injecting pairs of AAV vectors carrying separate fragments of Otoferlin DNA into the round window membrane to the affected cochlea. When these AAV vectors recombine, they produce the expression of the full-length gene and restores hearing. Dual AAV gene therapy provides a biologically regenerative treatment that is faster and less invasive than the cochlear implant currently used to treat DFNB9. This breakthrough will reshape the treatment of genetic diseases.
Title: | NOVEL GENE THERAPY TECHNIQUE TO TREAT HEREDITARY DEAFNESS. |
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Name(s): |
Mc Comie, Myca-Lee, author Wetterer, James K., Thesis advisor Florida Atlantic University Harriet L. Wilkes Honors College |
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Type of Resource: | text | |
Genre: | Thesis | |
Date Created: | 2020 | |
Date Issued: | 2020 | |
Publisher: | Florida Atlantic University Digital Library | |
Place of Publication: | Boca Raton, Fla. | |
Physical Form: | online resource | |
Extent: | 19 p. | |
Language(s): | English | |
Abstract/Description: | Autosomal recessive deafness-9 (DFNB9), caused by mutations in the otoferlin gene (OTOF), is the most common form of hereditary deafness, accounting for 2-8% of all cases. Here, I review recent research on using dual adeno associated virus (AAV) mediated gene therapy to treat DFNB9 in a mouse model system. Dual AAV gene therapy repairs these mutations by injecting pairs of AAV vectors carrying separate fragments of Otoferlin DNA into the round window membrane to the affected cochlea. When these AAV vectors recombine, they produce the expression of the full-length gene and restores hearing. Dual AAV gene therapy provides a biologically regenerative treatment that is faster and less invasive than the cochlear implant currently used to treat DFNB9. This breakthrough will reshape the treatment of genetic diseases. | |
Identifier: | FA00013663 (IID) | |
Degree granted: | Thesis (B.A.)--Florida Atlantic University, Harriet L. Wilkes Honors College, 2020. | |
Collection: | Florida Atlantic University Digital Library Collections | |
Note(s): | Includes bibliography. | |
Persistent Link to This Record: | http://purl.flvc.org/fau/fd/FA00013663 | |
Use and Reproduction: | Copyright © is held by the author with permission granted to Florida Atlantic University to digitize, archive and distribute this item for non-profit research and educational purposes. Any reuse of this item in excess of fair use or other copyright exemptions requires permission of the copyright holder. | |
Use and Reproduction: | http://rightsstatements.org/vocab/InC/1.0/ | |
Host Institution: | FAU |