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publicationDate 2021-07-08-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2021207138-A1
titleOfInvention Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
abstract The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5′-GUGGCUAACAGAAGCU (SEQ ID NO:1), 5′-GGGAACAUGCUAAAUAC (SEQ ID NO:2), 5′-AGACACAAAUUCCUGAGA (SEQ ID NO:3), or 5′-CUGUUGAGAAA (SEQ ID NO. 4), and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
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