Harvard College

The Brigham and Women's Hospital, Inc.

Children's Medical Center Corporation

Joslin Diabetes Center, Inc.

The United States of America, as Represented by the Secretary, Department of Health and Human Services

The General Hospital Corporation

Amy J Wagers

Lee L Rubin

Amanda Gee

Richard Theodore Lee

Leonard I Zon

George M Church

Mohammadsharif Tabebordbar

Feodor D Price

Wei Leong Chew

Amanda K W Gee

Jill Goldstein

Sahar Tavakoli

C Ronald Kahn

James Pancoast

Francesco Loffredo

Analise Z Reeves

Cammie Lesser

Ryan G Walker

Cong Xu

Ya-Chieh Hsu

Leo Wang

Matthew Steinhauser

Meryem Gonzalez Celeiro

Irene Min

Lida Katsimpardi

IN SITU GENE EDITING

Methods of editing target nucleic acids are provided using a guide RNA and a Cas9 protein to excise exons in a target gene and where the edited gene is expressed to produce a truncated polypeptide.

RNA-guided systems for in vivo gene editing

Disclosed herein are agents that enhance muscle stem cell engraftment, as well as methods and compositions using the same.

Methods and agents that enhance myogenic progenitor cell engraftment

The invention provides methods for inducing, enhancing or increasing satellite cell proliferation, and an assay for screening for a candidate compound for inducing, enhancing or increasing satellite cell proliferation. Also provided are methods for repairing or regenerating a damaged muscle tissue of a subject.

Small molecules for mouse satellite cell proliferation

Disclosed herein are GDF11 variant polypeptides. Also disclosed herein are methods for increasing GDF11 protein levels in a subject by administering a GDF11 variant polypeptide.

GDF11 variants and uses thereof

Methods and compositions for rejuvenating skeletal muscle stem cells are disclosed.

Methods and compositions for rejuvenating skeletal muscle stem cells

Methods of increasing satellite cell proliferation with vorinostat or bosutinib

Method of increasing satellite cell proliferation with an HDAC inhibitor

The methods and compositions described herein relate to the delivery of polypeptides to a target cell, e.g. by utilizing engineered non-pathogenic bacteria comprising a type three secretion system (T3SS) and T3SS-compatible substrates. In one aspect, described herein are non-pathogenic microbial cells that have been engineered to express both a functional type three secretion system (T3SS) and at least one polypeptide that is compatible with the T3SS. Due to the wide variety of polypeptides that can be delivered to a target eukaryotic cell using the compositions and systems described herein, a commensurately wide variety of applications is contemplated, e.g. therapeutics and reprogramming.

Compositions and methods for bacterial delivery of polypeptides

Methods of increasing satellite cell proliferation with kinase inhibitors

Disclosed are methods of inducing differentiation of stem into myogenic cells without gene manipulation and for inducing proliferation of satellite cells. The cells can be used as a source of cells for transplantation in a subject in need thereof. Also disclosed is a screening assay for screening test compounds using blastomere cultures.

High-throughput image-based chemical screening in zebrafish blastomere cell culture

Growing community of inventors

Cambridge, MA, United States of America

Amy J Wagers