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Gene therapy for the treatment of X-linked retinitis pigmentosa
INTRODUCTION: X-linked retinitis pigmentosa caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene is the most common form of recessive RP. The phenotype is characterised by its severity and rapid disease progression. Gene therapy using adeno-associated viral vectors is current...
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| 發表在: | Expert Opin Orphan Drugs |
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| Main Authors: | , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
2018
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6059358/ https://ncbi.nlm.nih.gov/pubmed/30057863 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/21678707.2018.1444476 |
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