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OLIGONUCLEOTIDE THERAPY FOR LEBER CONGENITAL AMAUROSIS
OLIGONUCLEOTIDE THERAPY FOR LEBER CONGENITAL AMAUROSIS
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机译:脱糖核苷酸治疗Leber先天性阿颈病
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摘要
Antisense oligonucleotides target the mutation in introit 26 of the CEP290 gene and reduce inclusion of the aberrant exon into the CEP290 mRNA. The oligonucleotides include no more than 3 consecutive guanosines, have no more than 60% guanosine nucleobases, include at most one CpG sequence, and/or do not have the potential to form a hairpin comprising 3 or more consecutive complementary base pairs.
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