Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis
Author(s) -
Amy Leung,
Almudena Sacristán-Reviriego,
Pedro R.L. Perdigão,
Sai Hali,
Michalis Georgiou,
Angelos Kalitzeos,
AmandaJayne F. Carr,
Peter Coffey,
Michel Michaelides,
James Bainbridge,
Michael E. Cheetham,
Jacqueline van der Spuy
Publication year - 2022
Publication title -
stem cell reports
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.207
H-Index - 76
ISSN - 2213-6711
DOI - 10.1016/j.stemcr.2022.08.005
Subject(s) - biology , organoid , retinal , induced pluripotent stem cell , microbiology and biotechnology , retina , cancer research , embryonic stem cell , genetics , neuroscience , gene , biochemistry
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