Pathophysiology of Human Blood CellsDescription of Major Goals
Goal of this program is to train physicians who will be the future academic leaders in pediatric Hematology/Oncology.
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell DiseaseDescription of Major Goals
The goal is to move to Phase 2 trial targeting BCL11A in SCD
Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell Disease - OTADescription 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.
Targeting and transfection of hematopoietic stem cellsDescription of Major Goals
Goal of this award is to develop methods for efficient in vivo gene therapy targeting SCD
SPORE in Myeloid Malignancies - Core 3Description 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.
Disseminating Curative Biological Therapies for Rare Pediatric DiseasesDescription 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.
Gene therapy for SCID-X1 with low dose busulfan and a SIN-lentiviral vectorDescription 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.
Gene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell DiseaseDescription 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.
HSCI FACS Core Facility AwardDescription 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.
A phase I/II, non randomized, multicenter, open-label study of G1XCGD (lentiviral vector transduced CD34+cells) in patients with x-linked chronic granulomatous diseaseDescription of Major Goals
Purpose of the study is to treat children with x-linked chronic granulomatous disease.
Translational and clinical studies targeting γ-globin modulationDescription 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.
Gene Transfer for SCID-X1 using a self-inactivating gammaretroviral vectorDescription 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.
Rac proteins in hematopoietic cell survival and functionDescription of Major Goals
The major goal of this project is to determine the role of Rac GTPase signaling in blood cell development and function.