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Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce ABCA4 splice variants in Stargardt disease
Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Ida Maria Westin, Irina Golovleva, Guei-Sheung Liu, Satheesh Kumar, Samuel McLenachan, Fred Kuanfu Chen, Fei-Ting Hsu, Tom Edwards, Keith R Martin, Albert Wu Cheng, View ORCID ProfileRaymond C.B. Wong
doi: https://doi.org/10.1101/2024.03.08.584155
Roxanne Hsiang-Chi Liou
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
Daniel Urrutia-Cabrera
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
Ida Maria Westin
3Medical and Clinical Genetics, Department of Medical Biosciences, University of Umeå, Sweden
Irina Golovleva
3Medical and Clinical Genetics, Department of Medical Biosciences, University of Umeå, Sweden
Guei-Sheung Liu
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
Satheesh Kumar
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
Samuel McLenachan
4Centre for Ophthalmology and Visual Science, University of Western Australia, Australia
5Lions Eye Institute, Western Australia, Australia
Fred Kuanfu Chen
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
4Centre for Ophthalmology and Visual Science, University of Western Australia, Australia
5Lions Eye Institute, Western Australia, Australia
Fei-Ting Hsu
6Department of Biological Science and Technology, China Medical University, Taiwan
Tom Edwards
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
Keith R Martin
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia
7Department of Clinical Neurosciences, University of Cambridge, United Kingdom
Albert Wu Cheng
8School of Biological and Health Systems Engineering, Arizona State University, United States
9State Key Laboratory of Stem Cell and Reproductive Biology, Institute of Zoology, Chinese Academy of Sciences, Beijing, China
10Key Laboratory of Organ Regeneration and Reconstruction, Chinese Academy of Sciences, Beijing, China
11Beijing Institute for Stem Cell and Regenerative Medicine, Beijing, China
12University of Chinese Academy of Sciences, Beijing, China
Raymond C.B. Wong
1Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital. Australia
2Ophthalmology, Department of Surgery, University of Melbourne, Australia

Article usage
Posted March 09, 2024.
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce ABCA4 splice variants in Stargardt disease
Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Ida Maria Westin, Irina Golovleva, Guei-Sheung Liu, Satheesh Kumar, Samuel McLenachan, Fred Kuanfu Chen, Fei-Ting Hsu, Tom Edwards, Keith R Martin, Albert Wu Cheng, Raymond C.B. Wong
bioRxiv 2024.03.08.584155; doi: https://doi.org/10.1101/2024.03.08.584155
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce ABCA4 splice variants in Stargardt disease
Roxanne Hsiang-Chi Liou, Daniel Urrutia-Cabrera, Ida Maria Westin, Irina Golovleva, Guei-Sheung Liu, Satheesh Kumar, Samuel McLenachan, Fred Kuanfu Chen, Fei-Ting Hsu, Tom Edwards, Keith R Martin, Albert Wu Cheng, Raymond C.B. Wong
bioRxiv 2024.03.08.584155; doi: https://doi.org/10.1101/2024.03.08.584155
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