Dana-Farber/Boston Children's Cancer and Blood Disorders Center
2022
Boston
MA
Publications
ANKRD26-related Thrombocytopenia 2 with a Baseline Increase in Blasts: Implications for Clinical Surveillance. View Abstract
Transcription factor networks disproportionately enrich for heritability of blood cell phenotypes. View Abstract
Inherited resilience to clonal hematopoiesis by modifying stem cell RNA regulation. View Abstract
CEBPA repression by MECOM blocks differentiation to drive aggressive leukemias. View Abstract
Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia. View Abstract
Transcription factor networks disproportionately enrich for heritability of blood cell phenotypes. View Abstract
A noncoding regulatory variant in IKZF1 increases acute lymphoblastic leukemia risk in Hispanic/Latino children. View Abstract
Editorial: Advances in predisposition to bone marrow failure and hematopoietic neoplasms. View Abstract
Deciphering cell states and genealogies of human haematopoiesis. View Abstract
High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia. View Abstract
High-Content Small Molecule Screen Identifies a Novel Compound That Restores AP-4-Dependent Protein Trafficking in Neuronal Models of AP-4-Associated Hereditary Spastic Paraplegia. View Abstract
Massively parallel base editing to map variant effects in human hematopoiesis. View Abstract
A genetic disorder reveals a hematopoietic stem cell regulatory network co-opted in leukemia. View Abstract
Familial thrombocytopenia due to a complex structural variant resulting in a WAC-ANKRD26 fusion transcript. View Abstract