- Patent Title: Methods and compositions for treating skeletal muscular dystrophy
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Application No.: US16605489Application Date: 2018-04-18
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Publication No.: US11759482B2Publication Date: 2023-09-19
- Inventor: Eduardo Marban , Mark Amin Aminzadeh , Russell Rogers , Jennifer Moseley , Luis Rodriguez-Borlado , Saravana Kanagavelu , Christopher Stewart Sakoda
- Applicant: Cedars-Sinai Medical Center , Capricor, Inc
- Applicant Address: US CA Los Angeles
- Assignee: Cedars-Sinai Medical Center,Capricor, Inc.
- Current Assignee: Cedars-Sinai Medical Center,Capricor, Inc.
- Current Assignee Address: US CA Los Angeles; US CA Beverly Hills
- Agency: Knobbe, Martens, Olson & Bear, LLP
- International Application: PCT/US2018/028184 2018.04.18
- International Announcement: WO2018/195210A 2018.10.25
- Date entered country: 2019-10-15
- Main IPC: A61K35/34
- IPC: A61K35/34 ; A61P25/14 ; A61P21/00 ; A61K31/7105 ; C12N15/113 ; A61K9/00

Abstract:
Some embodiments provide a method of treating skeletal muscular myopathy, e.g., Duchenne muscular dystrophy (DMD), with cardiosphere-derived cells (CDCs), wherein a therapeutically effective amount of CDCs is delivered to a targeted dystrophic skeletal muscle. Some embodiment enable delivery of a therapeutically effective amount of CDCs via intramuscular injection directly at a skeletal muscle or systemic administration, intravenous injection, in a single dose or multiple doses, to treat a targeted dystrophic skeletal muscle. Some embodiments provide a method for improving exercise capabilities in DMD patients. Additional embodiments relate to exosome, mediated transfer of noncoding RNAs ameliorates Duchenne muscular dystrophy by restoring dystrophin in heart and skeletal muscle. Delivery of noncoding RNA species found in CDC-derived exosomes mimics the ability of CDCs and CDC-derived exosomes to increase dystrophin protein levels.
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