Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_0414d2771e954bbeb16e2b74dd52a684 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-11 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-315 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-321 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-344 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-346 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3521 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2320-33 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3231 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-3525 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-113 |
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 |