公开(公告)号：US20220306573A1

公开(公告)日：2022-09-29

申请号：US17046752

申请日：2019-04-11

Applicant: Jason Jingxi ZHANG ,  Chandra VARGEESE ,  Naoki IWAMOTO ,  Chikdu Shakti SHIVALILA ,  Nayantara KOTHARI ,  Ann Fiegen DURBIN ,  Selvi RAMASAMY ,  Pachamuthu KANDASAMY ,  Jayakanthan KUMARASAMY ,  Gopal Reddy BOMMINENI ,  Subramanian MARAPPAN ,  Sethumadhavan DIVAKARAMENON ,  David Charles Donnel BUTLER ,  Genliang LU ,  Hailin YANG ,  Mamoru SHIMIZU ,  Prashant MONIAN ,  WAVE LIFE SCIENCES LTD.

Inventor： Jason Jingxin Zhang ,  Chandra Vargeese ,  Naoki Iwamoto ,  Chikdu Shakti Shivalila ,  Nayantara Kothari ,  Ann Fiegen Durbin ,  Selvi Ramasamy ,  Pachamuthu Kandasamy ,  Jayakanthan Kumarasamy ,  Gopal Reddy Bommineni ,  Subramanian Marappan ,  Sethumadhavan Divakaramenon ,  David Charles Donnell Butler ,  Genliang Lu ,  Hailin Yang ,  Mamoru Shimizu ,  Prashant Monian

IPC: C07C317/28 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113

Abstract: Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for reducing levels of transcripts. In some embodiments, the present disclosure provides technologies useful for modulating transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.