Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_2a7ebd2ce5ef7e691bd44c48a88a2bfc http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_b12f0d44fedff92f970fb40a547058be |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2800-80 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2810-10 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K2039-53 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K2039-515 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-10 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-34 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-907 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N9-1205 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N5-0696 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N5-0658 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-074 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N15-90 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N9-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K35-34 |
filingDate |
2015-05-18-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ecd961fe0f6111f8704a0bf73a7d8da7 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_bffd3491ec8ff65079cbd0189a317790 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_5804a73231c1a9a7ba8c82f192696290 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_d004420bdb5359f66f987f3febf8e294 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_cec87921cc3fdf76aa7346134370a8d4 |
publicationDate |
2017-03-30-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
US-2017088819-A1 |
titleOfInvention |
Genetic correction of myotonic dystrophy type 1 |
abstract |
The invention relates to polynucleotides suitable for reducing or eliminating the expression of expanded repeat RNA (CUGexp) of the dystrophy myotonic-protein kinase (DMPK) gene in a cell of a DM-1 patient. The polynucleotides are a combination of a polynucleotide for a site specific nuclease targeting the dystrophy myotonic-protein kinase (DMPK) gene locus, and a donor polynucleotide having 5′ and 3′ regions which are homologous with the sequence of DMPK gene which flank the target site of the nuclease. The invention further relate to in vivo and in vitro methods to reduce or eliminate CTG repeats in the DMPK gene. The invention further relates to the medical use of polynucleotides and cells for treating DM-1 patient. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10335466-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10920227-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10570395-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10597660-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11603542-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11542506-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11759506-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11198873-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11104897-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2019256868-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2018100144-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10584337-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11193129-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021076883-A3 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11752181-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11027000-B2 |
priorityDate |
2014-05-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |