Research & Laboratories
at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
The next frontier for innovation, research, clinical trials, and state-of-the art treatment.
Our history of research and innovation has resulted in many of the diagnostic and therapeutic techniques currently used around the world to treat cancer and blood disorders. Today, we continue to stand among the world’s leaders in many innovative approaches.
Certain genetic disorders cause widespread disease in the body, but the principle reason for illness and death in early childhood is failure of the blood system.
The Armstrong Lab studies how developmental gene expression programs are regulated during normal and cancer development.
We are leveraging cutting-edge protein engineering methods to create genetically-encodable macromolecules with novel function.
We are identifying genomic and epigenetic drivers of pediatric brain tumors, characterizing resistance mechanisms of pediatric brain tumors, and developing more effective treatments.
The laboratory studies the genetic determinants of blood cell ontogeny and disease. We use molecular genetic, biochemical, and genome editing methodologies to perturb and observe blood cells.
We study outcome disparities in childhood cancer with a focus on improving childhood cancer outcomes by systematically considering social determinants of health as risk factors in the clinical trial setting and potential targets for intervention.
The Camargo laboratory focuses on the study of adult stem cell biology, organ size regulation, and cancer.
We strive to apply cutting-edge technologies to address these problems and currently utilize a variety of approaches including proteomics, genomics, chromatin interaction analysis, gene editing, mouse genetics, human genetics, and human iPS cell technology.
CENTER FOR PLATELET RESEARCH STUDIES
The Center for Platelet Research Studies is an internationally recognized multidisciplinary center for the study of platelet function by state-of-the-art methods.
The Crompton Lab’s research focuses on utilizing genomic and proteomic technologies to identify and validate new therapeutic targets for pediatric solid tumors.
The D'Andrea lab investigates chromosome instability and susceptibility to cancer. Our laboratory examines the molecular signaling pathways which regulate the DNA damage response in mammalian cells.
Our laboratory seeks a better understanding of the biology, pathology, and clinical utility of hematopoietic and pluripotent stem cells and of the role of various tissue stem cells in development and disease.
The Filbin Lab at the Dana-Farber Cancer Institute studies pediatric brain tumors, particularly the lethal high-grade gliomas including DIPG and malignant embryonal brain tumors that are in greatest need of therapeutic improvements.
We are a team of young and eclectic scientists interested in exploiting gene-engineering tools to study biological functions and solve problems with a direct impact on human health.
We seek to identify molecular targets that can be translated into novel therapies in the pediatric solid tumor neuroblastoma, as well as to unravel the genetic perturbations that occur during development of the sympathetic nervous system and underlie neuroblastoma initiation and progression.
GOESSLING AND NORTH LABS
Using the zebrafish model for chemical fishing expeditions, we examine conserved regulators of stem cell specification and growth, ultimately striving towards clinical therapies to alleviate human hematopoietic and hepatic disease.
Our research is focused on identifying and characterizing new mechanisms of RNA regulation in the dynamic control of gene expression.
The primary focus of the Gutierrez lab is to define the molecular mechanisms underlying resistance to cancer therapies, and to translate these findings into novel therapeutic strategies.
Our laboratory is centered in understanding the structure and function of large, macromolecular machines called ATP-dependent chromatin remodeling complexes, with an emphasis in dissecting their roles in human disease and identifying new therapeutic opportunities.
Dr. Kean’s laboratory is working to solve the mystery of immune recognition and immune tolerance.
We are focused on the analysis of intratumoral heterogeneity in different cancer types, as well as the interactions between tumor cells and their microenvironment.
The broad interest of the Kim Lab is to characterize the biology of stem cells in normal lung and lung cancer.
Our research has helped defined the structure, interactions, and assembly-disassembly mechanisms of clathrin and many of its associated proteins, through studies extending over three decades.
Our research efforts focus on identifying epigenetic dependencies in pediatric tumors with a particular focus on high risk pediatric leukemias and sarcomas.
Our laboratory seeks to elucidate the molecular pathogenesis of human leukemias and solid tumors using both a cell culture model and a zebrafish animal model.
The Orkin laboratory focuses on stem cell biology, particularly the development and function of the blood system, the relationship between cancer and stem cells, and the mechanisms responsible for self-renewal of stem cells and the switch from fetal to adult hemoglobin.
Our laboratory uses a combination of genetics, biochemistry and live-cell imaging to study cell division and the maintenance of genome stability.
Our goal is to use genomics and metabolomics to understand pediatric leukemia biology and develop strategies to integrate targeted therapies for treatment of leukemia.
We are interested in unlocking new paradigms of normal blood development with the long-term aim of building better models of childhood blood diseases.
Our goal is to understand how genetic variation alters human blood and immune cell production, or hematopoiesis, in health and disease.
Our research focuses on intercellular communication: how growth factor signaling pathways regulate brain development, and how these pathways contribute to abnormal proliferation, migration and survival in brain tumors and in other neurologic disorders.
The Shimamura lab studies bone marrow failure, myelodysplastic syndromes, and genetic cancer predisposition.
We study receptor-ligand interactions and transmembrane signal transmission that are relevant to immunology, hemostasis, and human disease using structural, cell biological, and single molecule techniques.
Our research program focuses on the integration of "omic" approaches for the identification of new protein targets and small-molecule modulators of malignancy with an eye toward clinical translation.
Our mission is to promote basic and applied discovery in the fields of vascular biology, inflammation, and thrombosis.
The Walensky Lab focuses on the chemical biology of deregulated apoptotic and transcriptional pathways in cancer.
The Weinstock Laboratory uses a range of approaches at the intersection of genetics, DNA repair, and mouse modeling to address the ontogeny, pathogenesis, and therapeutic targeting of lymphoid neoplasms.
The Williams Lab has focused on understanding the interaction of hematopoietic stem cells with the bone marrow and abnormalities of these interactions, which are associated with leukemia.
Our research program is quite diverse, ranging from classical cellular immunology using mouse models, studies in the human system and structural biology, over viral infection models and cell biology using live microscopy, to molecular biology and global epigenetic analyses.
Our research seeks to impact care throughout the illness trajectory from diagnosis, advanced illness, end-of-life, and bereavement, and across all ages from perinatal through young adults.
Our lab investigates how biological systems work at the nanoscale, and the physical laws that govern their behavior. We are particularly interested in how mechanical forces regulate biological processes ranging from hearing to bleeding.
Over the last decade, the Zhang lab has made significant contributions to the epigenetic field by identifying and characterizing many chromatin modifying enzymes that include: 1) the nucleosome remodeling and deacetylase NuRD; 2) the H3K27me3 methyltransferase PRC2; 3) the ubiquitin E3 ligase PRC1; 4) the JmjC histone demethylases; and 5) the Tet family of 5mC dioxygenases and novel nucleotides 5fC and 5caC.
Our mission is to discover the principles governing blood cell production and melanoma to guide the development of therapies for blood disorders & cancer
Our Researchers in the News
Avoiding a lifetime of injections: Can gene editing cure severe congenital neutropenia?
Fionn Mulrooney, a cheerful 11-month-old, in Plymouth, Massachusetts, has no idea he has a life-threatening genetic disease. Nor does he seem fazed by the daily subcutaneous injections his parents have learned how to give him. And little does he know that cells from his bone marrow are helping scientists develop an innovative gene-editing approach that could someday correct his disease, known as severe congenital neutropenia or SCN.
Looking for cancer’s Achilles heel: The Pediatric Cancer Dependency Map
Thanks to developments in precision medicine, some adult cancers are now treated with designer drugs that target the genetic mutations that caused them. But most children with cancer have not reaped the same benefits. Unlike adult cancers, childhood cancers carry few genetic mutations. And the mutations these tumors do have are typically harder to make drugs against.
After decades of evolution, gene therapy arrives
As early as the 1960s, scientists speculated that DNA sequences could be introduced into patients’ cells to cure genetic disorders. In the early 1980s, David Williams, MD, and David Nathan, MD, at Boston Children’s Hospital published the first paper showing one could use a virus to insert genes into blood-forming stem cells. In 2003, the Human Genome Project wrapped up, giving us a complete blueprint of our DNA. In the past decade, gene therapy has become a reality for multiple diseases, especially those caused by mutations in a single gene.