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Active Grants and Sponsored Research

Project Date: January 1, 2018 - December 31, 2022
Sponsoring Organization: St Jude's Children's Research Hospital

Novel Gene Therapies for Sickle Cell Diseases

Description of Major Goals
High-throughput discovery of essential noncoding sequences for erythropoiesis The goals of this project are to utilize high-throughput genome editing technology and knowledge of human genetic variation associated with erythroid traits to characterize noncoding sequences required for erythropoiesis. The intention is to develop improved models of noncoding sequence function by iterative experimental testing and analytic refinement.
Sponsor Grant ID: 2P01HL032262-35A1 Project Date: April 16, 2017 - March 31, 2022
Sponsoring Organization: National Heart, Lung, and Blood Institute

Developmental Biology of Human Erythropoiesis

Description of Major Goals
The goals of the project “Functional Dissection of Erythroid Super-Enhancers” is to investigate the sequence requirements and trans-acting factors interacting with critical erythroid super-enhancer elements. The proposal integrates genome editing, bioinformatic, and biochemistry methodologies to explore fundamental mechanisms of erythroid gene regulation.
Sponsor Grant ID: DP2 HL137300 Project Date: September 30, 2016 - June 30, 2021
Sponsoring Organization: National Heart, Lung, and Blood Institute

High-Throughput Discovery of Essential Noncoding Sequences for Erythtropoiesis

Description of Major Goals
The goals of this project are to utilize high-throughput genome editing technology and knowledge of human genetic variation associated with erythroid traits to characterize noncoding sequences required for erythropoiesis. The intention is to develop improved models of noncoding sequence function by iterative experimental testing and analytic refinement.
Project Date: September 1, 2017 - August 31, 2020
Sponsoring Organization: Doris Duke Charitable Foundation

Small molecule targeted reactivation of HbF for sickle cell disease

Description of Major Goals
The goal of this project is to transform management of SCD through the development of new drug candidates specifically targeted to interfere with fetal hemoglobin (HbF) silencing. The aims are: Perform comprehensive, fine-resolution functional CRISPR/Cas9 mapping of NuRD complex components; Develop small molecules targeting the bromodomain of ZMYND8; Assess the consequences of ZMYND8 bromodomain domain inhibitors and directed destruction of ZMYND8 protein for HbF induction; and assess the functional consequences of small molecules directed to ZMYND8 for HbF reactivation, and other gene expression and cellular changes, in both wild-type and SCD-patient derived primary CD34 cells.
Project Date: September 1, 2015 - August 31, 2020
Sponsoring Organization: Harvard Medical School - Burroughs Wellcome Fund

Functional Characterization of Trait Associated Enhancers

Description of Major Goals
The goals of this research are to synthesize genetics, biology, and technology to test the hypothesis that enhancer variation is a prevailing determinant of human traits. We will systematically disrupt blood cell trait-associated enhancers to investigate impact on blood cell development, the genetic architecture of blood cell traits, and define cis- and trans-acting determinants of hematopoiesis.