http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2017088819-A1

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filingDate 2015-05-18-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ecd961fe0f6111f8704a0bf73a7d8da7
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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
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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
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http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021076883-A3
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11752181-B2
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priorityDate 2014-05-16-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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Total number of triples: 632.