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Pathogenic mechanisms and the prospect of gene therapy for choroideremia
INTRODUCTION: Choroideremia is a rare, X-linked disorder recognized by its specific ocular phenotype as a progressive degenerative retinopathy resulting in blindness. New therapeutic approaches, primarily based on genetic mechanisms, have emerged that aim to prevent the progressive vision loss. AREA...
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| Publicado en: | Expert Opin Orphan Drugs |
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| Autores principales: | , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
2015
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4522943/ https://ncbi.nlm.nih.gov/pubmed/26251765 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1046434 |
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