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NOVEL GENE THERAPY TECHNIQUE TO TREAT HEREDITARY DEAFNESS

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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
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

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