公开(公告)号：US20160160217A1

公开(公告)日：2016-06-09

申请号：US15042371

申请日：2016-02-12

Applicant: University of Massachusetts

Inventor： Charles P. Emerson ,  Jennifer Chen ,  Oliver D. King

IPC: C12N15/113

CPC classification number: C12N15/113 ,  A01K67/0271 ,  C12N5/0658 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/12 ,  C12N2320/30 ,  C12Q1/6883 ,  C12Q2600/106 ,  C12Q2600/158 ,  C12N2310/3525

Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.

Abstract translation: 用于鉴定面积性营养不良肌营养不良症（FSHD）的新疗法的组合物和方法及其用途。

公开(公告)号：US09260755B2

公开(公告)日：2016-02-16

申请号：US13861227

申请日：2013-04-11

Applicant: University of Massachusetts

Inventor： Charles P. Emerson, Jr. ,  Jennifer Chen ,  Oliver D. King

IPC: A01K67/00 ,  C12Q1/68 ,  C12N15/113 ,  C12N15/11 ,  A01K67/027 ,  C12N5/077

CPC classification number: C12N15/113 ,  A01K67/0271 ,  C12N5/0658 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/12 ,  C12N2320/30 ,  C12Q1/6883 ,  C12Q2600/106 ,  C12Q2600/158 ,  C12N2310/3525

Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.

Abstract translation: 用于鉴定面积性营养不良肌营养不良症（FSHD）的新疗法的组合物和方法及其用途。

公开(公告)号：US20130347136A1

公开(公告)日：2013-12-26

申请号：US13861227

申请日：2013-04-11

Applicant: University of Massachusetts

Inventor： Charles P. Emerson, JR. ,  Jennifer Chen ,  Oliver D. King

IPC: C12Q1/68 ,  A01K67/027 ,  C12N5/077

CPC classification number: C12N15/113 ,  A01K67/0271 ,  C12N5/0658 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/12 ,  C12N2320/30 ,  C12Q1/6883 ,  C12Q2600/106 ,  C12Q2600/158 ,  C12N2310/3525

Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.

Abstract translation: 用于鉴定面积性营养不良肌营养不良症（FSHD）的新疗法的组合物和方法及其用途。

公开(公告)号：US20210230568A1

公开(公告)日：2021-07-29

申请号：US17051632

申请日：2019-05-03

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Scot A. Wolfe ,  Charles P. Emerson, JR. ,  Sukanya Iyer ,  Sneha Suresh ,  Christian Mueller ,  Jennifer Chen ,  Dongsheng Guo ,  Oliver King

IPC: C12N9/22 ,  A61K35/76 ,  A61K38/00

Abstract: The present invention is directed to the filed of gene therapy. In particular, compositions and methods are disclosed that repair gene microduplication mutations by reversion to a wild type sequence. For example, the creation of a double stranded break by a programmable nuclease protein within a microduplication induces the microhomology mediated end joining DNA repair pathway that in the process of DNA repair removes the microduplication mutation and restores the wild type sequence.