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Active Grants and Sponsored Research

Sponsor Grant ID: 5T32HL007574-40 Project Date: July 1, 2020 - June 30, 2025
Sponsoring Organization: National Heart, Lung And Blood Institute (NHLBI)

Pathophysiology of Human Blood Cells

Description of Major Goals
Goal of this program is to train physicians who will be the future academic leaders in pediatric Hematology/Oncology.
Sponsor Grant ID: CLIN2SCD-12031 Project Date: January 1, 2021 - December 31, 2024
Sponsoring Organization: CALIFORNIA INSTITUTE OF REGENERATIVE MED

Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease

Description of Major Goals
The goal is to move to Phase 2 trial targeting BCL11A in SCD
Sponsor Grant ID: 1OT2HL154815-01 Project Date: July 22, 2020 - December 31, 2024
Sponsoring Organization: NIH/NHLBI

Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease - OTA

Description of Major Goals
Project goal is to extend patient enrollment accrual target for phase 1 trial and move to Phase 2 trial targeting BCL11A in SCD.
Sponsor Grant ID: INV-021791 Project Date: September 1, 2020 - August , 2022
Sponsoring Organization: BILL AND MELINDA GATES FOUNDATION

Targeting and transfection of hematopoietic stem cells

Description of Major Goals
Goal of this award is to develop methods for efficient in vivo gene therapy targeting SCD
Sponsor Grant ID: 7P50CA206963-05 Project Date: April 1, 2019 - July 31, 2022
Sponsoring Organization: NIH/NCI

SPORE in Myeloid Malignancies - Core 3

Description of Major Goals
Conduction of BH3 profiling assays for the leukemia SPORE investigators. The assay provides a predictive biomarker that can help guide the use of approved therapies for multiple cancer indications.
Sponsor Grant ID: 1U01TR001814-01 Project Date: September 15, 2016 - June 30, 2022
Sponsoring Organization: NIH/NCATS

Disseminating Curative Biological Therapies for Rare Pediatric Diseases

Description of Major Goals
This project seeks to develop a network of pediatric centers with unique expertise and experience in translation of gene therapies. Our overall goal is to support investigators across multiple CTSAs to more rapidly translate complex gene therapies to early phase investigator-initiated pediatric clinical trials suitable for transfer to industry.
Sponsor Grant ID: 5U01AI125051-06 Project Date: July 1, 2016 - June 30, 2022
Sponsoring Organization: NIH/NIAID

Gene therapy for SCID-X1 with low dose busulfan and a SIN-lentiviral vector

Description of Major Goals
This project seeks to test the efficacy and safety of a new self-inactivating lentiviral (LV) vector to treat SCIDX1. We hypothesize that this trial will improve immune reconstitution through the introduction of low dose busulfan conditioning and improve safety through the change from a gammaretroviral vector used in previous trials to the LV vector in this trial.
Sponsor Grant ID: 1R01HL137848-05 Project Date: September 5, 2017 - May 31, 2022
Sponsoring Organization: NIH/NHLBI

Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease

Description of Major Goals
Induction of fetal hemoglobin (HbF) in sickle cell disease (SCD) is an extremely promising approach to ameliorate the severity of this disease. Decreasing the expression of BCL11A in sickle cell patients could increase the amount of fetal hemoglobin, while simultaneously reducing the amount of sickle hemoglobin, in their blood and therefore potentially cure the condition. In this clinical trial, we will use a virus vector to reduce the expression of this gene in red blood cells.
Sponsor Grant ID: CF-0007-14-00 Project Date: July 1, 2010 - September 30, 2021
Sponsoring Organization: Harvard Stem Cell Institute

HSCI FACS Core Facility Award

Description of Major Goals
This grant is to support a core facility that isolates normal and leukemia stem cells for investigators a Boston Children’s Hospital and the Harvard Stem Cell Institute.
Sponsor Grant ID: 1649-S-TA394 Project Date: May 1, 2015 - August 31, 2021
Sponsoring Organization: CALIFORNIA INSTITUTE OF REGENERATIVE MED

A phase I/II, non randomized, multicenter, open-label study of G1XCGD (lentiviral vector transduced CD34+cells) in patients with x-linked chronic granulomatous disease

Description of Major Goals
Purpose of the study is to treat children with x-linked chronic granulomatous disease.
Sponsor Grant ID: 5U01HL117720-05 Project Date: August 15, 2013 - May 31, 2019
Sponsoring Organization: National Institutes Of Health (NIH)

Translational and clinical studies targeting γ-globin modulation

Description of Major Goals
The overarching goal is to utilize innovative scientific approaches to identify and put into clinical investigation new therapeutic modalities that modulate γ-globin expression.
Sponsor Grant ID: 5U01AI087628-05 Project Date: September 01, 2010 - August 31, 2018
Sponsoring Organization: National Institute Of Allergy And Infect Diseases (NIAID)

Gene Transfer for SCID-X1 using a self-inactivating gammaretroviral vector

Description of Major Goals
The aims of this study are to assess the safety of somatic gene transfer for SCID-X1 using the pSRS11.EFS.IL2RG.pre* vector, assess the efficacy of somatic gene transfer in specific lineages, and assess the impact on T and B cell reconstitution and clinical outcome. My role as co-PI is to serve as co-investigator on the clinical trial, assist in setting up and implementation of the trial, recruit subjects, and supervise endpoint analysis in my laboratory.
Sponsor Grant ID: 5R01DK062757 Project Date: April 01, 2002 - June 30, 2017
Sponsoring Organization: National Institute Diab & Digest & Kid Diseases (NIDDKD)

Rac proteins in hematopoietic cell survival and function

Description of Major Goals
The major goal of this project is to determine the role of Rac GTPase signaling in blood cell development and function.