Nature Communications (Apr 2022)
In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration
- Elliot H. Choi,
- Susie Suh,
- Andrzej T. Foik,
- Henri Leinonen,
- Gregory A. Newby,
- Xin D. Gao,
- Samagya Banskota,
- Thanh Hoang,
- Samuel W. Du,
- Zhiqian Dong,
- Aditya Raguram,
- Sajeev Kohli,
- Seth Blackshaw,
- David C. Lyon,
- David R. Liu,
- Krzysztof Palczewski
Affiliations
- Elliot H. Choi
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- Susie Suh
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- Andrzej T. Foik
- International Centre for Translational Eye Research, Polish Academy of Sciences
- Henri Leinonen
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- Gregory A. Newby
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Xin D. Gao
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Samagya Banskota
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Thanh Hoang
- Department of Neuroscience, Johns Hopkins University School of Medicine
- Samuel W. Du
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- Zhiqian Dong
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- Aditya Raguram
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Sajeev Kohli
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Seth Blackshaw
- Department of Neuroscience, Johns Hopkins University School of Medicine
- David C. Lyon
- Department of Anatomy and Neurobiology, School of Medicine, University of California
- David R. Liu
- Merkin Institute of Transformative Technologies in Healthcare at Broad Institute
- Krzysztof Palczewski
- Gavin Herbert Eye Institute, Department of Ophthalmology, University of California
- DOI
- https://doi.org/10.1038/s41467-022-29490-3
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 14
Abstract
Leber congenital amaurosis is caused by mutations in RPE65 and leads to retinal degeneration in children. Here, the authors show that in vivo base editing can prolong the survival of cone photoreceptors and rescue their function in a mouse model of the disease.
