公开(公告)号：US20200031890A1

公开(公告)日：2020-01-30

申请号：US16593840

申请日：2019-10-04

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Julian Ramos ,  Stephen D. Hauschka

IPC: C07K14/47 ,  C07H21/04 ,  A61K48/00 ,  A61P21/00 ,  C12N15/85

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

公开(公告)号：US20170362635A1

公开(公告)日：2017-12-21

申请号：US15628533

申请日：2017-06-20

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Niclas Bengtsson ,  Stephen D. Hauschka

IPC: C12Q1/68 ,  C07H21/02 ,  C12N15/11 ,  C12N15/63 ,  C12N9/22 ,  C07H21/04 ,  A61K38/43 ,  C12N15/10 ,  C12N15/00 ,  C12N2310/20

CPC classification number: C12Q1/68 ,  A61K38/43 ,  C07H21/02 ,  C07H21/04 ,  C12N9/22 ,  C12N15/00 ,  C12N15/102 ,  C12N15/111 ,  C12N15/113 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/32 ,  C12N2330/51

Abstract: Pharmaceutical compositions including a muscle-specific nuclease cassette, one or more guide RNA cassettes, and a delivery system for delivery of the muscle-specific nuclease cassette and the one or more gRNA cassettes are provided. The pharmaceutical composition may also include a mutation-corrected DNA template including a modification to be made in a target nucleic acid sequence. Methods for treating a subject having a muscular or neuromuscular disorder are also provided. The methods may include administering to the subject a therapeutically effective amount of the pharmaceutical composition. Methods of modifying or editing the sequence of a target nucleic acid sequence in a muscle cell are also provided. The methods may include contacting or transducing the muscle cell with a muscle-specific nuclease cassette and one or more gRNA cassettes.

公开(公告)号：US20180148488A1

公开(公告)日：2018-05-31

申请号：US15541870

申请日：2016-01-15

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Julian Ramos ,  Stephen D. Hauschka

IPC: C07K14/47 ,  A61P21/00 ,  A61K48/00 ,  C12N15/85

CPC classification number: C07K14/4708 ,  A61K38/00 ,  A61K48/0058 ,  A61P21/00 ,  C07H21/04 ,  C07K14/4707 ,  C07K14/4716 ,  C07K2319/30 ,  C12N15/85 ,  C12N2750/14143 ,  C12N2830/008

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

公开(公告)号：US11958888B2

公开(公告)日：2024-04-16

申请号：US16593833

申请日：2019-10-04

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Julian Ramos ,  Stephen D. Hauschka

IPC: C07K14/47 ,  A61K38/00 ,  A61K48/00 ,  A61P21/00 ,  C07H21/04 ,  C12N15/85 ,  C12N15/86

CPC classification number: C07K14/4708 ,  A61K48/0058 ,  A61P21/00 ,  C07H21/04 ,  C07K14/4707 ,  C07K14/4716 ,  C12N15/85 ,  C12N15/86 ,  A61K38/00 ,  C07K2319/30 ,  C12N2750/14143 ,  C12N2830/008

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

公开(公告)号：US20200095298A1

公开(公告)日：2020-03-26

申请号：US16593833

申请日：2019-10-04

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Julian Ramos ,  Stephen D. Hauschka

IPC: C07K14/47 ,  C07H21/04 ,  A61K48/00 ,  A61P21/00 ,  C12N15/85

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.

公开(公告)号：US20240200093A1

公开(公告)日：2024-06-20

申请号：US18286215

申请日：2022-04-07

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Stephen D. Hauschka

IPC: C12N15/86

CPC classification number: C12N15/86 ,  C12N2750/14143 ,  C12N2830/008

Abstract: A library of artificial muscle-specific expression cassettes (MSECs) for muscle-specific gene expression is described. Different members of the library can be selected for varied transcription levels in different muscle cell types, for different research or therapeutic purposes. MSECs within the library can be used to develop treatments for muscle-related disorders.

公开(公告)号：US10479821B2

公开(公告)日：2019-11-19

申请号：US15541870

申请日：2016-01-15

Applicant: University of Washington

Inventor： Jeffrey S. Chamberlain ,  Julian Ramos ,  Stephen D. Hauschka

IPC: C07K14/47 ,  A61K48/00 ,  A61P21/00 ,  C12N15/85 ,  A61K38/00

Abstract: Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.