Art

J-GLOBAL ID：201902284365773034   Reference number：19A0494642

Correction of Monogenic and Common Retinal Disorders with Gene Therapy

遺伝子治療による単遺伝子性および一般性網膜疾患の補正【JST・京大機械翻訳】

Author (12)： Sengillo Jesse D. (Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY 10032, USA) ,  Sengillo Jesse D. (Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA) ,  Sengillo Jesse D. (State University of New York Downstate Medical Center, Brooklyn, NY 11203, USA) ,  Justus Sally (Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY 10032, USA) ,  Justus Sally (Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA) ,  Cabral Thiago (Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY 10032, USA) ,  Cabral Thiago (Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA) ,  Cabral Thiago (Department of Ophthalmology, Federal University of Espirito Santo, Vitoria 29075-910, Brazil) ,  Cabral Thiago (Department of Ophthalmology, Federal University of Sao Paulo, Sao Paulo 04021-001, Brazil) ,  Tsang Stephen H. (Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY 10032, USA) ,  Tsang Stephen H. (Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA) ,  Tsang Stephen H. (Department of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA)
Material： Genes (Web)
Volume： 8  Issue： 2  Page： 53  Publication year： 2017 
JST Material Number： U7194A  ISSN： 2073-4425  Document type： Article
Article type： 文献レビュー  Country of issue： Switzerland (CHE)  Language： ENGLISH (EN)

Abstract/Point： The past decade has seen major...

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Thesaurus term： lens(optics) ,  *gene therapy ,  *retinal disease ,  accessibility ,  *heredity ,  *therapy ,  *universality ,  *gene ,  clinical trial
Semi thesaurus term： 最適性 ,  ベッドサイド ,  動物モデル ,  非侵襲 ,  免疫特権 , 【Automatic Indexing@JST】

Author keywords (1)： gene therapy genome engineering CRISPR retinal degeneration inherited retinal disease

JST classification (3)： Treatment for eye diseases in general  (GP05010T) ,  Eye diagnosis(= ophthalmic diagnosis)  (GP02000N) ,  Eye diseases  (GP03000U)

Reference (179)：

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• Kohn, D.B.; Sadelain, M.; Glorioso, J.C. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat. Rev. Cancer 2003, 3, 477-488.
• Bennett, J.; Tanabe, T.; Sun, D.; Zeng, Y.; Kjeldbye, H.; Gouras, P.; Maguire, A.M. Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy. Nat. Med. 1996, 2, 649-654.
• Acland, G.M.; Aguirre, G.D.; Ray, J.; Zhang, Q.; Aleman, T.S.; Cideciyan, A.V.; Pearce-Kelling, S.E.; Anand, V.; Zeng, Y.; Maguire, A.M.; et al. Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet. 2001, 28, 92-95.
• Bainbridge, J.W.; Smith, A.J.; Barker, S.S.; Robbie, S.; Henderson, R.; Balaggan, K.; Viswanathan, A.; Holder, G.E.; Stockman, A.; Tyler, N.; et al. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N. Engl. J. Med. 2008, 358, 2231-2239.

Terms in the title (4)： 遺伝子治療 ,  遺伝子 ,  一般性 ,  網膜疾患