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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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 AT xuetingluo progressinresearchonlca2genetherapy AT weitan progressinresearchonlca2genetherapy |
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