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Analysis of a large choroideremia dataset does not suggest a preference for inclusion of certain genotypes in future trials of gene therapy
BACKGROUND: Choroideremia (CHM) is an X‐linked degeneration of the retinal pigment epithelium, photoreceptors, and choroid, which causes nyctalopia and progressive constriction of visual fields leading to blindness. The CHM gene encodes Rab escort protein 1 (REP‐1). In this work, we reviewed the phe...
Autores principales: | Freund, Paul R., Sergeev, Yuri V., MacDonald, Ian M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4867567/ https://www.ncbi.nlm.nih.gov/pubmed/27247961 http://dx.doi.org/10.1002/mgg3.208 |
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