A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration
نویسندگان
چکیده
منابع مشابه
Gene therapy for inherited retinal degeneration.
Research into the molecular and genetic basis of disease is continually expanding and improving the prospects for rational treatments. Of these, gene therapy (here defined as the introduction of genetic material into human cells) may ultimately oVer the greatest scope. This article provides a perspective on the potential for gene therapy for treating inherited retinal degenerations, along with ...
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PURPOSE To describe the genotype-phenotype correlation and serial observations in a five-generation Czech family with X-linked retinitis pigmentosa (XLRP) associated with severe visual impairment in women. METHODS Comprehensive ophthalmological examination including spectral domain optical coherence tomography (SD-OCT) was performed. Based on the pedigree structure and women being severely af...
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ژورنال
عنوان ژورنال: Human Molecular Genetics
سال: 2015
ISSN: 1460-2083,0964-6906
DOI: 10.1093/hmg/ddv134