Gene replacement therapy restores RCBTB1 expression and cilium length in patient‐derived retinal pigment epithelium

Journal of Cellular and Molecular Medicine

Sue Fletcher¹  Samuel McLenachan²  Livia S. Carvalho²  Zhiqin Huang²  Fred K. Chen³  Dan Zhang⁴  Shang‐Chih Chen⁴  Luke Jennings⁴ 

[1] Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Murdoch, WA, Australia;Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, Nedlands, WA, Australia;Centre for Ophthalmology and Visual Science, The University of Western Australia, Perth, WA, Australia;Lions Eye Institute, Nedlands, WA, Australia;Centre for Ophthalmology and Visual Science, The University of Western Australia, Perth, WA, Australia;Lions Eye Institute, Nedlands, WA, Australia;Department of Ophthalmology, Royal Perth Hospital, Perth, WA, Australia;Department of Ophthalmology, Perth Children’s Hospital, Nedlands, WA, Australia;Lions Eye Institute, Nedlands, WA, Australia;

关键词: adeno‐associated virus;    gene therapy;    induced pluripotent stem cells;    inherited retinal disease;    RCBTB1;    retinal pigment epithelium;   

DOI  :  10.1111/jcmm.16911

来源: Wiley

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