In:
Science Translational Medicine, American Association for the Advancement of Science (AAAS), Vol. 14, No. 654 ( 2022-07-20)
Abstract:
Targeting disease-relevant transcripts using RNA editing holds promise for treating genetic diseases, without the risks associated with permanent changes induced by DNA alterations. Here, Xiao et al. evaluated the therapeutic potential of a Cas13-derived RNA base editor for correcting a hearing loss–causing mutation in the myosin VI ( Myo6 ) transcript in a mouse model. The RNA base editor composed of a Cas13X variant and the RNA editing enzyme adenosine deaminase was delivered in the cochlea of mice using an AAV. The treatment prevented hair cell loss and rescued auditory function in mice for up to 3 months after injection, suggesting that RNA editing might be effective for treating genetic disorders.
Type of Medium:
Online Resource
ISSN:
1946-6234
,
1946-6242
DOI:
10.1126/scitranslmed.abn0449
Language:
English
Publisher:
American Association for the Advancement of Science (AAAS)
Publication Date:
2022
Permalink