Active Grants and Sponsored Research
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
Development of novel selective Rac inhibitors for refractory leukemias
Description of Major GoalsThe goal of this project is to carry out orthogonal approaches to develop small molecule inhibitors of Rac for the treatment of refractory leukemias (in particular, MLL-rearranged and RAS-mutated).
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: 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: 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: 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.