Progress in research on LCA2 gene therapy

Leber's congenital amaurosis(LCA)is a genetic eye disease that can cause blindness. Infants with LCA may have a severe low vision or loss of vision at the early stage. The LCA2 type of this disease is related to RPE65 mutation. According to previous studies, there is no effective treatment for...

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Main Authors: Ru-Yi Zhao, Xue-Ting Luo, Wei Tan
Format: Article
Language:English
Published: Press of International Journal of Ophthalmology (IJO PRESS) 2019-10-01
Series:Guoji Yanke Zazhi
Subjects:
Online Access:http://ies.ijo.cn/cn_publish/2019/10/201910015.pdf
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spelling doaj-77288b5c557d47628de67164329130872020-11-25T01:32:42ZengPress of International Journal of Ophthalmology (IJO PRESS)Guoji Yanke Zazhi1672-51231672-51232019-10-0119101700170310.3980/j.issn.1672-5123.2019.10.15Progress in research on LCA2 gene therapyRu-Yi Zhao0Xue-Ting Luo1Wei Tan2Department of Ophthalmology, the Third Affiliated Hospital of Zunyi Medical University, Zunyi 563000, Guizhou Province, ChinaDepartment of Ophthalmology, Shanghai First Hospital, Shanghai 200080, ChinaDepartment of Ophthalmology, the Third Affiliated Hospital of Zunyi Medical University, Zunyi 563000, Guizhou Province, ChinaLeber's congenital amaurosis(LCA)is a genetic eye disease that can cause blindness. Infants with LCA may have a severe low vision or loss of vision at the early stage. The LCA2 type of this disease is related to RPE65 mutation. According to previous studies, there is no effective treatment for genetic retinal diseases including LCA2. In recent years, with the advances in gene therapy technology, great progress in the treatment of genetic retinal diseases has been made, among which the most successful one is the gene therapy of LCA2. This paper briefly introduces the development of the gene therapy of LCA2, and reviews the correlation between age and injection type, dosage, injection method, measuring method as well as therapeutic effect and the stability of therapeutic effect in previous clinic trials, which provides reference and clinical treatment experience for the clinical application of the gene therapy of LCA2 in China.http://ies.ijo.cn/cn_publish/2019/10/201910015.pdfLeber congenital amaurosisLCA2RPE65gene therapyhereditary retinal disease
collection DOAJ
language English
format Article
sources DOAJ
author Ru-Yi Zhao
Xue-Ting Luo
Wei Tan
spellingShingle Ru-Yi Zhao
Xue-Ting Luo
Wei Tan
Progress in research on LCA2 gene therapy
Guoji Yanke Zazhi
Leber congenital amaurosis
LCA2
RPE65
gene therapy
hereditary retinal disease
author_facet Ru-Yi Zhao
Xue-Ting Luo
Wei Tan
author_sort Ru-Yi Zhao
title Progress in research on LCA2 gene therapy
title_short Progress in research on LCA2 gene therapy
title_full Progress in research on LCA2 gene therapy
title_fullStr Progress in research on LCA2 gene therapy
title_full_unstemmed Progress in research on LCA2 gene therapy
title_sort progress in research on lca2 gene therapy
publisher Press of International Journal of Ophthalmology (IJO PRESS)
series Guoji Yanke Zazhi
issn 1672-5123
1672-5123
publishDate 2019-10-01
description Leber's congenital amaurosis(LCA)is a genetic eye disease that can cause blindness. Infants with LCA may have a severe low vision or loss of vision at the early stage. The LCA2 type of this disease is related to RPE65 mutation. According to previous studies, there is no effective treatment for genetic retinal diseases including LCA2. In recent years, with the advances in gene therapy technology, great progress in the treatment of genetic retinal diseases has been made, among which the most successful one is the gene therapy of LCA2. This paper briefly introduces the development of the gene therapy of LCA2, and reviews the correlation between age and injection type, dosage, injection method, measuring method as well as therapeutic effect and the stability of therapeutic effect in previous clinic trials, which provides reference and clinical treatment experience for the clinical application of the gene therapy of LCA2 in China.
topic Leber congenital amaurosis
LCA2
RPE65
gene therapy
hereditary retinal disease
url http://ies.ijo.cn/cn_publish/2019/10/201910015.pdf
work_keys_str_mv AT ruyizhao progressinresearchonlca2genetherapy
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