Advances in rAAV in the Treatment of Hereditary Deafness
WEI Ding
BAI Xinyue
DU Jiani
WANG Zhuoling
WANG Qingyan
CHEN Yahui
ZHAI Xiaoyan
Abstract：According to the World Health Organization 2021,more than 50％of congenital hearing loss is caused by genetic variants of factors.Several of the main clinical modalities used to improve hearing in hearing loss patients(e.g.,hearing aids,cochlear implants)have limitations.Recombination adeno-associated virus(rAAV)has been widely used as a relatively safe gene therapy vector in clinical and basic research of genetic diseases.The application of single rAAV in the research and treatment of diseases caused by defects in large gene fragments is limited due to the restricted size of the packaging vector.Currently,teams have developed dual rAAVs for the treatment of diseases caused by defects in large gene fragments.This review highlights the progress of dual rAAV in deafness diseases caused by defective large gene fragments to help develop more gene therapy rAAV for deafness diseases in the future.
Keywords：genetic deafnessrecombination adeno-associated virusdual recombination adeno-associated virusgene therapy
Publication Date：2024-10-28
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：7( 861-867 )