OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF

Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use GC thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be...

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Bibliographische Detailangaben
Hauptverfasser: RAMASAMY, Selvi, MARAPPAN, Subramanian, BUTLER, David Charles Donnell, KOTHARI, Nayantara, SHIVALILA, Chikdu Shakti, MONIAN, Prashant, IWAMOTO, Naoki, KUMARASAMY, Jayakanthan, BOMMINENI, Gopal Reddy, DIVAKARAMENON, Sethumadhavan, DURBIN, Ann Fiegen, ZHANG, Jason Jingxin, SHIMIZU, Mamoru, VARGEESE, Chandra, LU, Genliang, KANDASAMY, Pachamuthu, YANG, Hailin
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Zusammenfassung:Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use GC thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.