Applications of CRISPR/Cas9 in retinal degenerative diseases
| Autor: | Luo Sheng Tang, Ying Qian Peng, Yedi Zhou, Shigeo Yoshida |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
Retinal degeneration Gene therapy of the human retina business.industry Cas9 Genetic enhancement Review medicine.disease Bioinformatics 03 medical and health sciences Ophthalmology 030104 developmental biology Genome editing Retinitis pigmentosa medicine CRISPR business Induced pluripotent stem cell |
| Popis: | Gene therapy is a potentially effective treatment for retinal degenerative diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has been developed as a new genome-editing tool in ophthalmic studies. Recent advances in researches showed that CRISPR/Cas9 has been applied in generating animal models as well as gene therapy in vivo of retinitis pigmentosa (RP) and leber congenital amaurosis (LCA). It has also been shown as a potential attempt for clinic by combining with other technologies such as adeno-associated virus (AAV) and induced pluripotent stem cells (iPSCs). In this review, we highlight the main points of further prospect of using CRISPR/Cas9 in targeting retinal degeneration. We also emphasize the potential applications of this technique in treating retinal degenerative diseases. |
| Databáze: | OpenAIRE |
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