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filingDate 2016-02-26-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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publicationDate 2021-12-09-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber AU-2016223336-B2
titleOfInvention Oligonucleotide therapy for leber congenital amaurosis
abstract Antisense oligonucleotides target the mutation in intron 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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