http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2022127610-A1

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filingDate 2020-02-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_5cb01817a8c0517540d0ae3ee74c850e
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0d205cb713a5f3432ca04f9393ffec20
publicationDate 2022-04-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2022127610-A1
titleOfInvention Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa
abstract The invention relates to antisense oligonucleotides (AONs) and the use thereof in the treatment of human disease. In particular the present invention is concerned with AONs suitable for the treatment of Dystrophic Epidermolysis Bullosa (DEB). More specifically, the invention relates to AONs that are capable of inducing exon 105 skipping from human COL7A1 pre-mRNA and that outperform—in exon 105 skipping efficiency—the AONs known from the prior art. The invention further relates to the use of these new AONs in the treatment of DEB in a patient carrying a mutation in exon 105 of the COL7A1 gene.
priorityDate 2019-02-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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