SKELETAL MYOBLAST PROGENITOR CELL LINEAGE SPECIFICATION BY CRISPR/CAS9-BASED TRANSCRIPTIONAL ACTIVATORS

Title:

SKELETAL MYOBLAST PROGENITOR CELL LINEAGE SPECIFICATION BY CRISPR/CAS9-BASED TRANSCRIPTIONAL ACTIVATORS

Document Type and Number:

WIPO Patent Application WO/2021/034984

Kind Code:

A3

Abstract:

Disclosed herein are methods and systems for increasing expression of Pax7, methods of activating endogenous myogenic transcription factor Pax7 in a cell, methods of differentiating a stem cell into a skeletal muscle progenitor cell, as well as compositions and methods for treating a subject in need of regenerative muscle progenitor cells. The compositions and methods may include a Cas9-based transcriptional activator protein and at least one guide RNA (gRNA) targeting Pax7.

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Inventors:

GERSBACH CHARLES (US)
KWON JENNIFER (US)

Application Number:

PCT/US2020/047080

Publication Date:

April 01, 2021

Filing Date:

August 19, 2020

Export Citation:

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Assignee:

UNIV DUKE (US)

International Classes:

A61K48/00; A61P21/00; C12N15/113; C12N15/85

Domestic Patent References:

WO2016130600A2

2016-08-18

Foreign References:

US20170204407A1

2017-07-20

Attorney, Agent or Firm:

COX, Julia, M. et al. (US)

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