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.
Development of novel selective Rac inhibitors for refractory leukemiasDescription of Major Goals
The 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).
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.
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.
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.