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Active Grants and Sponsored Research

Sponsor Grant ID: 1R35HG010717-01 Project Date: September 1, 2019 - June 30, 2024
Sponsoring Organization: NIH

Multiscale exploration of the functional non-coding genome

Description of Major Goals
The goal of this subproject is to plan, generate and analyze data related to base editing screening, allele enrichment by long-read sequencing and UMI incorporation, as well as single cell RNA sequencing following genome editing studies.
Sponsor Grant ID: 5R01HL150553-02 Project Date: January 1, 2020 - December 31, 2023
Sponsoring Organization: National Heart, Lung, and Blood Institute

Gene editing ELANE to understand and treat severe congenital neutropenia

Description of Major Goals
The goal is to apply novel gene editing technologies to develop a highly efficient, simple therapeutic approach to better understand and eventually treat ELANE-associated severe congenital neutropenia.
Sponsor Grant ID: 1R01HL150669-01 Project Date: December 20, 2019 - November 30, 2023
Sponsoring Organization: National Heart, Lung, and Blood Institute

Rectifying splicing mutations in blood disorders by gene editing

Description of Major Goals
The goal is to develop innovative gene editing methods that can repair the abnormal genes that cause Beta-thalassemia and Shwachman-Diamond syndrome to restore their normal processing.
Sponsor Grant ID: 5P01HL053749-22 Project Date: August 15, 2018 - June 30, 2023
Sponsoring Organization: NIH/NNLBI

Lentiviral Gene Therapy For Sickle Cell Disease and Immunodeficiency Disorders

Description of Major Goals
The goal of this project is to develop improved lentiviral gene therapy vectors for erythroid-specific expression of BCL11A shRNAs. Additionally, this project aims to compare two distinct approaches for de-repressing HbF in adult human red blood cells via genome editing.
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.
Project Date: October 1, 2020 - September 30, 2022
Sponsoring Organization: Harvard Medical School

Therapeutic gene editing of ELANE for severe congenital neutropenia

Description of Major Goals
The goal is to conduct preclinical development enabling a clinical trial of therapeutic gene editing for severe congenital neutropenia. We will: define the most favorable sites in ELANE for Cas9 editing to produce premature termination codons (PTCs); maximize Cas9 editing specifically, including evaluation by genome-wide off-target editing assessment; investigate impact on HSC function as measured by xenograft assay; and compare healthy donor and ELANE mutant CD34+ HSPCs.
Sponsor Grant ID: 2019116 Project Date: September 1, 2019 - August 30, 2022
Sponsoring Organization: Doris Duke Charitable Foundation

Enhanced nuclease delivery for therapeutic gene editing of hematopoietic stem cells in sickle cell disease

Description of Major Goals
The goal of this project is to develop improved nucleases for delivery to hematopoietic stem cells for therapeutic gene editing that obviates need for ex vivo cell electroporation.
Sponsor Grant ID: 1OT2HL154984-01 Project Date: June 18, 2020 - May 31, 2022
Sponsoring Organization: National Heart, Lung, and Blood Institute

Therapeutic BCL11A enhancer gene editing to induce fetal hemoglobin in Beta-hemoglobinopathy patients- CureSci

Description of Major Goals
The goal of this project is to conduct a clinical trial of therapeutic gene editing of the BCL11A erythroid enhancer in patients with sickle cell disease and beta-thalassemia to durably induce fetal hemoglobin.
Sponsor Grant ID: 5P01HL032262-39 Project Date: April 16, 2017 - March 31, 2022
Sponsoring Organization: National Heart, Lung, and Blood Institute

Developmental Biology of Human Erythropoiesis - Project 4

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.
Project Date: September 1, 2015 - August 31, 2021
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.
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.