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ANTISENSE OLIGONUCLEOTIDES FOR USE IN THE TREATMENT OF USHER SYNDROME
ANTISENSE OLIGONUCLEOTIDES FOR USE IN THE TREATMENT OF USHER SYNDROME
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机译:反义寡核苷酸用于治疗迎膜综合征
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摘要
The invention relates to the fields of medicine and to antisense oligonucleotides (AONs) that are capable of skipping exon 50 from human USH2A pre-mRNA and that may be used in the treatment, prevention and/or delay of Usher syndrome type II and/or USH2A-associated non syndromic retina degeneration.
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