Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease
Year: 2025
Type: article
Language: en
Topic: Retinal Development and Disorders
Authors and affiliations:
raw_author_name: Roxanne Hsiang-Chi Liou
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Daniel Urrutia-Cabrera
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Chia-Fei Liu
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Department of Health Technology and Informatics, The Hong Kong Polytechnic University, Hong Kong, China
raw_author_name: Sihui Wu
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Ida Maria Westin
raw_affiliation_strings: Medical and Clinical Genetics, Department of Medical Biosciences, Umeå University, Umeå, Sweden
raw_author_name: Irina Golovleva
raw_affiliation_strings: Medical and Clinical Genetics, Department of Medical Biosciences, Umeå University, Umeå, Sweden
raw_author_name: Guei-Sheung Liu
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Satheesh Kumar
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Samuel McLenachan
raw_affiliation_strings: Centre for Ophthalmology and Visual Science, University of Western Australia, Perth, Western Australia, Australia
Lions Eye Institute, Perth, Western Australia, Australia
raw_author_name: Fred Kuanfu Chen
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Centre for Ophthalmology and Visual Science, University of Western Australia, Perth, Western Australia, Australia
Lions Eye Institute, Perth, Western Australia, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Fei-Ting Hsu
raw_affiliation_strings: Department of Life Sciences, National Central University, Taoyuan City, Taiwan
raw_author_name: Chien-Ling Huang
raw_affiliation_strings: Department of Health Technology and Informatics, The Hong Kong Polytechnic University, Hong Kong, China
raw_author_name: Tom Edwards
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Keith R. Martin
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia
raw_author_name: Albert Wu Cheng
raw_affiliation_strings: Beijing Institute for Stem Cell and Regenerative Medicine, Beijing, China
School of Biological and Health Systems Engineering, Arizona State University, Tempe, Arizona, USA
State Key Laboratory of Organ Regeneration and Reconstruction, Institute of Zoology, Chinese Academy of Sciences, Beijing, China
University of Chinese Academy of Sciences, Beijing, China
raw_author_name: Raymond C.B. Wong
raw_affiliation_strings: Centre for Eye Research Australia, Melbourne, Victoria, Australia
Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, Victoria, Australia