Children's Medical Center Corporation

The General Hospital Corporation

Dana-farber-cancer Institute Inc.

Harvard College

Joslin Diabetes Center, Inc.

Children's Medical Center Coporation

Leonard I Zon

Wolfram Goessling

Trista E North

Alison M Taylor

Pulin Li

Amy J Wagers

Adriana Donovan

Jun Qi

Dennis Buckley

George Q Daley

Craig Ceol

Elizabeth Macari

Yariv J Houvras

Joseph Mandelbaum

Sahar Tavakoli

Richard M White

Alan J Davidson

James Elliott Bradner

James Bradner

C Ronald Kahn

James Amatruda

Jennifer Shepard

Jennifer Best

Sadhana Agarwal

Brenda Vail

Paul Richardson

Elliott J Hagedorn

Cong Xu

Owen J Tamplin

Trista E N0rth

The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).

Calmodulin inhibitors for the treatment of ribosomal disorders and ribosomopathies

Described herein are methods for inducing HSC specification in a cell. Aspects of the invention relate to contacting a cell with a Runx1-CBFβ inhibitor for a specified period of time, and then removing the inhibitor from the cell. In some embodiments of any of the aspects, HSC specificity is maintained long-term.

Methods for inducing hematopoietic stem cell specificity

The technology described herein relates to compositions and methods of generating endothelial niche cells. Embodiments of the technology described herein comprise compositions, kits, vectors, and methods related to generating or engineering endothelial niche cells. One aspect comprises a method to generate/engineer endothelial niche cells, comprising expressing one or more transcription factors in an endothelial cell, wherein the one or more transcription factors are from the Ets family, the Sox family, and/or the Nuclear Hormone (NHR) family.

Endothelial cell factors and methods thereof

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 present invention relates to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to the use of novel classes of compounds, i.e. inhibitors of RSK (p90S6K); inhibitors of p70S6K; and inhibitors of rps6, to treat ribosomal disorders and ribosomopathies. In some embodiments, the invention relates to the use of specific Chk2 inhibitors and to the use of specific phenothiazine derivatives to treat ribosomal disorders and ribosomopathies, e.g. DBA.

Calmodulin inhibitors, Chk2 inhibitors and RSK inhibitors for the treatment of ribosomal disorders and ribosomapathies

Calmodulin inhibitors for the treatment of ribosomal disorders and ribosomapathies

The present invention provides for compositions and methods for modulating tissue growth using tissue growth modulators, which are agents that either enhance or inhibit tissue growth as desired by a particular indication by modulating the PG or Wnt signaling pathways, or employing modulators of both PG and Wnt signaling pathways for a synergistic effect or highly selective effect.

Method to enhance tissue regeneration

The present invention is directed to compositions and methods for treating Adenoid Cystic Carcinoma (ACC).

Methods for treatment of adenoid cystic carcinoma

Embodiments of the present invention are directed to methods for treatment of melanoma using an inhibitor of dihydroorotate dehydrogenase (DHODH) and to combination therapies that involve administering to a subject an inhibitor of oncogenic BRAF (e.g. BRAF(V600E)), as well as an inhibitor of dihydroorotate dehydrogenase (DHODH). Assays for identifying compounds useful for the treatment of melanoma are also provided. The methods comprise screening for compounds or agents that inhibit neural crest progenitor formation in a zebra fish model of melanoma.

Methods for treatment of melanoma

The present invention provides for compositions and methods for modulating hematopoetic stem cell populations by using HCS modulators, which are agents that either increase HSC numbers or decrease HSC numbers as desired by a particular indication. For example, HSC modulators found to increase HSC numbers include prostaglandin E(PGE2) and agents that stimulate the PGE2 pathway. Conversely, HSC modulators that prevent PGE2 synthesis decrease HSC numbers. HCS modulators may be used in vitro, in vivo, or ex vivo.

Methods for promoting hematopoietic reconstitution

Methods for promoting HSC engraftment

Growing community of inventors

Wellesley, MA, United States of America

Leonard I Zon