Active Grants and Sponsored Research
Sponsoring Organization: National Heart, Lung And Blood Institute (NHLBI)
Pathophysiology of Human Blood Cells
Description of Major GoalsGoal of this program is to train physicians who will be the future academic leaders in pediatric Hematology/Oncology.
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 GoalsThe goal is to move to Phase 2 trial targeting BCL11A in SCD
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 GoalsProject goal is to extend patient enrollment accrual target for phase 1 trial and move to Phase 2 trial targeting BCL11A in SCD.
Sponsoring Organization: BILL AND MELINDA GATES FOUNDATION
Targeting and transfection of hematopoietic stem cells
Description of Major GoalsGoal of this award is to develop methods for efficient in vivo gene therapy targeting SCD
Sponsoring Organization: NIH/NCI
SPORE in Myeloid Malignancies - Core 3
Description of Major GoalsConduction 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.
Sponsoring Organization: NIH/NCATS
Disseminating Curative Biological Therapies for Rare Pediatric Diseases
Description of Major GoalsThis 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.
Sponsoring Organization: NIH/NIAID
Gene therapy for SCID-X1 with low dose busulfan and a SIN-lentiviral vector
Description of Major GoalsThis 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.
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 GoalsInduction 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.
Sponsoring Organization: Harvard Stem Cell Institute
HSCI FACS Core Facility Award
Description of Major GoalsThis 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.
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 GoalsPurpose of the study is to treat children with x-linked chronic granulomatous disease.
Sponsoring Organization: National Institutes Of Health (NIH)
Translational and clinical studies targeting γ-globin modulation
Description of Major GoalsThe overarching goal is to utilize innovative scientific approaches to identify and put into clinical investigation new therapeutic modalities that modulate γ-globin expression.
Sponsoring Organization: National Institute Of Allergy And Infect Diseases (NIAID)
Gene Transfer for SCID-X1 using a self-inactivating gammaretroviral vector
Description of Major GoalsThe 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.
Sponsoring Organization: National Institute Diab & Digest & Kid Diseases (NIDDKD)
Rac proteins in hematopoietic cell survival and function
Description of Major GoalsThe major goal of this project is to determine the role of Rac GTPase signaling in blood cell development and function.