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METHODS AND COMPOSITIONS FOR TREATING LEBER CONGENITAL AMAUROSIS
METHODS AND COMPOSITIONS FOR TREATING LEBER CONGENITAL AMAUROSIS
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机译:治疗先天性耳聋的方法和成分
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摘要
Expression vectors, viral particles and therapeutic methods of using such constructs to improve the visual function of a patient suffering from diseases of the eye, resulting from failure to produce a specific protein in the eye, or the production of a non-functional protein in the eye, particularly Leber Congenital Amaurosis (LCA) and CEP290-related LCA.
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