http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-11066668-B2

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filingDate 2019-05-24-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2021-07-20-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_cd537964e3061d6efc837df4f567c1b7
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_df77a88d847ab25f8d6214d946ce3e9b
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publicationDate 2021-07-20-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-11066668-B2
titleOfInvention Compositions and methods for modulation of IKBKAP splicing
abstract The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.
priorityDate 2012-01-11-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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Total number of triples: 389.