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Targeted Gene Delivery into the Mammalian Inner Ear Using Synthetic Serotypes of Adeno-Associated Virus Vectors

Authors
 Min-A Kim  ;  Nari Ryu  ;  Hye-Min Kim  ;  Ye-Ri Kim  ;  Byeonghyeon Lee  ;  Tae-Jun Kwon  ;  Jinwoong Bok  ;  Un-Kyung Kim 
Citation
 MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, Vol.13 : 197-204, 2019 
Journal Title
 MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT 
Issue Date
2019
Keywords
adeno-associated virus ; gene therapy ; genetic hearing loss ; inner ear ; serotype
Abstract
Targeting specific cell types in the mammalian inner ear is important for treating genetic hearing loss due to the different cell type-specific functions. Adeno-associated virus (AAV) is an efficient in vivo gene transfer vector, and it has demonstrated promise for treating genetic hearing loss. Although more than 100 AAV serotypes have been identified, few studies have investigated whether AAV can be distributed to specific inner ear cell types. Here we screened three EGFP-AAV reporter constructs (serotypes DJ, DJ8, and PHP.B) in the neonatal mammalian inner ear by injection via the round window membrane to determine the cellular specificity of the AAV vectors. Sensory hair cells, supporting cells, cells in Reissner's membrane, interdental cells, and root cells were successfully transduced. Hair cells in the cochlear sensory epithelial region were the most frequently transduced cell type by all tested AAV serotypes. The recombinant DJ serotype most effectively transduced a range of cell types at a high rate. Our findings provide a basis for improving treatment of hereditary hearing loss using targeted AAV-mediated gene therapy.
Files in This Item:
T201901653.pdf Download
DOI
10.1016/j.omtm.2019.01.002
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Anatomy (해부학교실) > 1. Journal Papers
Yonsei Authors
Bok, Jin Woong(복진웅) ORCID logo https://orcid.org/0000-0003-1958-1872
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/170026
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