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Clinical applications of retinal gene therapies
Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (...
| Autores principales: | , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Oxford University Press
2018
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8982485/ https://www.ncbi.nlm.nih.gov/pubmed/35694125 http://dx.doi.org/10.1093/pcmedi/pby004 |
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| author | Fu, Xin Huu, Viet Anh Nguyen Duan, Yaou Kermany, Daniel S Valentim, Carolina C S Zhang, Runze Zhu, Jie Zhang, Charlotte L Sun, Xiaodong Zhang, Kang |
| author_facet | Fu, Xin Huu, Viet Anh Nguyen Duan, Yaou Kermany, Daniel S Valentim, Carolina C S Zhang, Runze Zhu, Jie Zhang, Charlotte L Sun, Xiaodong Zhang, Kang |
| author_sort | Fu, Xin |
| collection | PubMed |
| description | Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy. We review the history, present, and future prospects of retinal gene therapy. |
| format | Online Article Text |
| id | pubmed-8982485 |
| institution | National Center for Biotechnology Information |
| language | English |
| publishDate | 2018 |
| publisher | Oxford University Press |
| record_format | MEDLINE/PubMed |
| spelling | pubmed-89824852022-06-10 Clinical applications of retinal gene therapies Fu, Xin Huu, Viet Anh Nguyen Duan, Yaou Kermany, Daniel S Valentim, Carolina C S Zhang, Runze Zhu, Jie Zhang, Charlotte L Sun, Xiaodong Zhang, Kang Precis Clin Med Review Retinal degenerative diseases are a major cause of blindness. Retinal gene therapy is a trail-blazer in the human gene therapy field, leading to the first FDA approved gene therapy product for a human genetic disease. The application of Clustered Regularly Interspaced Short Palindromic Repeat/Cas9 (CRISPR/Cas9)-mediated gene editing technology is transforming the delivery of gene therapy. We review the history, present, and future prospects of retinal gene therapy. Oxford University Press 2018-06 2018-06-01 /pmc/articles/PMC8982485/ /pubmed/35694125 http://dx.doi.org/10.1093/pcmedi/pby004 Text en © The Author(s) 2018. Published by Oxford University Press on behalf of West China School of Medicine & West China Hospital of Sichuan University. https://creativecommons.org/licenses/by-nc/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) ), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com |
| spellingShingle | Review Fu, Xin Huu, Viet Anh Nguyen Duan, Yaou Kermany, Daniel S Valentim, Carolina C S Zhang, Runze Zhu, Jie Zhang, Charlotte L Sun, Xiaodong Zhang, Kang Clinical applications of retinal gene therapies |
| title | Clinical applications of retinal gene therapies |
| title_full | Clinical applications of retinal gene therapies |
| title_fullStr | Clinical applications of retinal gene therapies |
| title_full_unstemmed | Clinical applications of retinal gene therapies |
| title_short | Clinical applications of retinal gene therapies |
| title_sort | clinical applications of retinal gene therapies |
| topic | Review |
| url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8982485/ https://www.ncbi.nlm.nih.gov/pubmed/35694125 http://dx.doi.org/10.1093/pcmedi/pby004 |
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