Translation of CRISPR Genome Surgery to the Bedside for Retinal Diseases.
作者： Christine L. Xu , Galaxy Y. Cho , Jesse D. Sengillo , Karen S. Park , Vinit B. Mahajan
关键词:
摘要: In recent years, there has been accelerated growth of clustered regularly interspaced short palindromic repeats (CRISPR) genome surgery techniques. Genome holds promise for diseases which a cure currently does not exist. the field ophthalmology, CRISPR offers possibilities treating inherited retinal dystrophies. The retina little regenerative potential, makes treatment particularly difficult. For such conditions, methods have shown great potential therapeutic applications in animal models Much anticipation surrounds as therapeutic, clinical trials ophthalmic are expected to begin early 2018. This mini-review summarizes preclinical and current trials.
frontiersin.org
core.ac.uk
sci-hub.se 参考文章(40)
Hongxin Song, Ethan A. Rossi, Lisa Latchney, Angela Bessette, Edwin Stone, Jennifer J. Hunter, David R. Williams, Mina Chung, Cone and Rod Loss in Stargardt Disease Revealed by Adaptive Optics Scanning Light Ophthalmoscopy JAMA Ophthalmology. ,vol. 133, pp. 1198- 1203 ,(2015) , 10.1001/JAMAOPHTHALMOL.2015.2443
Yi-Ting Tsai, Stephen H. Tsang, Michael K. Lin, Emerging Treatments for Retinitis Pigmentosa: Genes and stem cells, as well as new electronic and medical therapies, are gaining ground. Retinal physician. ,vol. 12, pp. 52- 70 ,(2015)
Brian T. Fife, Kristen E. Pauken, The role of the PD-1 pathway in autoimmunity and peripheral tolerance. Annals of the New York Academy of Sciences. ,vol. 1217, pp. 45- 59 ,(2011) , 10.1111/J.1749-6632.2010.05919.X
Faheem Ahmed Khan, Muhammad Jamal, Lin Da, Zeshan Habib, Jinxia Dai, Gang Cao, Keeping CRISPR/Cas on-Target. Current Issues in Molecular Biology. ,vol. 20, pp. 1- 12 ,(2016)
Hua Zhong, Yiyun Chen, Yumei Li, Rui Chen, Graeme Mardon, CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes Scientific Reports. ,vol. 5, pp. 8366- 8366 ,(2015) , 10.1038/SREP08366
Prashant Mali, Luhan Yang, Kevin M Esvelt, John Aach, Marc Guell, James E DiCarlo, Julie E Norville, George M Church, RNA-Guided Human Genome Engineering via Cas9 Science. ,vol. 339, pp. 823- 826 ,(2013) , 10.1126/SCIENCE.1232033
Shengdar Q Tsai, Zongli Zheng, Nhu T Nguyen, Matthew Liebers, Ved V Topkar, Vishal Thapar, Nicolas Wyvekens, Cyd Khayter, A John Iafrate, Long P Le, Martin J Aryee, J Keith Joung, GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases Nature Biotechnology. ,vol. 33, pp. 187- 197 ,(2015) , 10.1038/NBT.3117
Sung Pyo Park, Jae Keun Chung, Vivienne Greenstein, Stephen H. Tsang, Stanley Chang, A study of factors affecting the human cone photoreceptor density measured by adaptive optics scanning laser ophthalmoscope Experimental Eye Research. ,vol. 108, pp. 1- 9 ,(2013) , 10.1016/J.EXER.2012.12.011
Jennifer A. Doudna, Emmanuelle Charpentier, The new frontier of genome engineering with CRISPR-Cas9 Science. ,vol. 346, pp. 1258096- 1258096 ,(2014) , 10.1126/SCIENCE.1258096
L. Cong, F. A. Ran, D. Cox, S. Lin, R. Barretto, N. Habib, P. D. Hsu, X. Wu, W. Jiang, L. A. Marraffini, F. Zhang, Multiplex Genome Engineering Using CRISPR/Cas Systems Science. ,vol. 339, pp. 819- 823 ,(2013) , 10.1126/SCIENCE.1231143