Alphabetical Listing of Laboratories Working on Rhabdo Research
For a Map of our Global Network of Rhabdomyosarcoma Research Laboratories – Click Here.
Click Here – Barr Lab
Bethesda, MD – Dr. Barr's research interest is molecular genetics of cancer with a focus on recurrent chromosomal alterations in sarcomas. His research team has most recently been conducting a multidisciplinary approach to understand the biological consequences of these genetic events in rhabdomyosarcoma, a family of pediatric soft tissue cancers. Major focal points of this research program are fusions of the PAX3 or PAX7 gene with the FOXO1 (FKHR) gene to generate fusion oncoproteins and the amplification of several oncogenic loci. These studies revealed that the gene fusions result in an aberrant gene expression program that contributes to tumorigenesis. Amplification events involving known and new candidate oncogenes occur in specific gene fusion subsets and are associated with distinct clinical outcomes. Current studies are examining tumorigenic effects, functional domains, downstream targets, regulation of gene expression, and clinical applications.
Click Here< – Chandler Lab
Columbus, OH – The Chandler lab focuses on the regulation of pre-mRNA splicing and its disruption leading to pediatric cancer and spinal muscular atrophy. We utilize cell culture models and mouse models to better understand the role of the alternative spliced forms as a manifestation of a disease.
Click Here – Davie Lab
Carbondale, IL – Research in my lab is focused on gene regulatory mechanisms in a development system. We focus on the specification and differentiation of skeletal muscle which is controlled by four highly related basic helix loop helix proteins referred to as the Myogenic Regulatory Factors, or MRFs. Much of the work in the lab is focused on understanding the function and specificity of myogenin, the only MRF singly required for viability. Our research is directed towards understanding the regulation of the genes controlled by myogenin by determining promoter elements that control expression and identifying additional protein factors that contribute to the unique role of myogenin on these genes, including chromatin modifying factors and additional transcription activators and co-activators.
Click Here – Alessandro Fanzani Lab
Brescia, Italy – The Fanzani lab currently focuses in establishing the functional role of the different caveolar proteins, termed Caveolins and Cavins, in the progression of rhabdomyosarcoma. These multifunctional proteins play a pivotal role in the generation of the plasma membrane domains known as caveolae, which are involved in endocytosis, cholesterol homeostasis and signal transduction. Understanding whether and how these proteins may facilitate or hamper the development of rhabdomyosarcoma is our main goal.
Click Here – Fulda Lab
Frankfurt, Germany – Apoptosis Signaling Pathways and Molecular Targeted Therapies – Professor of Experimental Cancer Research (W3), Director of the Institute for Experimental Cancer Research in Pediatrics
Click Here – The Galindo Lab
Dallas, Tx – The Galindo Lab is interested in the genetic and molecular underpinnings of childhood cancer. Within the group of malignant tumors that afflict children, those that grow from the skeleton and soft tissues (e.g, muscle, bone) are called sarcomas and are particularly aggressive.
Click Here – CRBM – Macromolecular Biochemestry Research Center – UMR 5237 – Cécile Gauthier-Rouvière
Montpellier, France – Rho GTPases, Adhesion and Physiopathology of Skeletal Muscle – – – Cécile Gauthier-Rouvière
Cell-cell adhesion molecules are essential for tissue organization and development. Equipe afiliée SBCFCadherin molecules act as key adhesion-activated signaling receptors important for many cellular processes, including myogenesis induction and fusion. We aim to identify the signaling pathways activated by the different cadherins expressed in skeletal muscle cells and in particular the Rho GTPase pathways regulating myogenesis as well as pathological development of rhabdomyosarcoma.
Click Here – Guttridge Lab
Columbus, OH – The Guttridge Lab at Ohio State investigates the role of the nuclear factor kappa-B (NF-κB) family of transcription factors in the growth and development of skeletal muscle. Recent lab findings have supported the idea that NF-κB functions as an inhibitor of cellular differentiation and have further elucidated the many mechanisms by which this occurs. Current projects in the lab are investigating the NF-κB signaling in myogenesis, rhabdomyosarcoma, Duchenne muscular dystrophy, and cancer cachexia.
Click Here – Hahn Lab
Göttingen, Germany – Cancer is a disease that results from inappropriate cell division induced by hyperproliferation. In many cases, the development of cancer is associated with genes or signaling pathways important for patterning during embryogenesis. We investigate the role of the Hedgehog/Patched (Hh/Ptch) signaling cascade in the development of solid tumors. The focus is on tumors caused by mutations in Ptch, such as medulloblastoma, rhabdomyosarcoma and basal cell carcinoma.
Click Here – Helman Lab
Bethesda, MD – Dr. Helman's laboratory currently focuses on three major themes related to the biology and treatment of pediatric sarcomas, specifically rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma: (1) determine the pathophysiologic consequences of IGF signaling; (2) identify the molecular/biochemical determinants of the biology of these sarcomas; and (3) apply preclinical laboratory findings to develop novel clinical studies for these sarcomas.
Click Here – Hollenbach Lab
New Orleans, LA – Dr. Hollenbach’s research focuses on understanding how phosphorylation of the transcription factor Pax3 regulates its biological activity to control muscle and melanocyte development and how alteration of this activity contributes to the development of the childhood solid muscle tumor alveolar rhabdomyosarcoma (ARMS) and melanoma. Towards this end, his lab has identified the three sites of phosphorylation on Pax3, they have identified two of the three kinases responsible for phosphorylating these sites, and they have determined that the pattern of Pax3 phosphorylation changes significantly during the first eight hours of myogenesis.
Click Here – Pediatric Preclinical Testing Program (PPTP) – Houghton
Columbus, OH – The Pediatric Preclinical Testing Program (PPTP) is a comprehensive program to systematically evaluate new agents against childhood solid tumor and leukemia models. The PPTP is supported through an NCI research contract to The Research Institute at Nationwide Children's Hospital with Dr. Peter Houghton as the Principal Investigator. Testing occurs both at The Research Institute and also at subcontract sites that have expertise in specific childhood cancers
Click Here – Charles Keller Laboratory at the Oregon Health and Science University
The Keller Laboratory studies the driving mechanisms and therapeutic targets in the childhood muscle cancers, alveolar rhabdomyosarcoma and embryonal rhabdomyosarcoma, and the childhood brain tumor, medulloblastoma.
Click Here – Khan Lab
Gaithersburg, MD – The mission of the Oncogenomics Section is to harness the power of high throughput genomic and proteomic methods to improve the outcome of children with high-risk metastatic, refractory and recurrent cancers. The research goals are to integrate the data, decipher the biology of these cancers and to identify and validate biomarkers and novel therapeutic targets and to rapidly translate our findings to the clinic.
Click Here – Marc Ladanyi Lab
New York City, NY – The research program in this laboratory focuses on the genomics and molecular pathogenesis of sarcomas and thoracic malignancies, with an emphasis on clinical translation of potential diagnostic markers and therapeutic targets. Dr. Ladanyi also co-directs (with Chris Sander) the Genome Data Analysis Center at Memorial Sloan-Kettering, which is part of the TCGA project network.
Click Here – Langenau Lab
Charlestown, MA – The Langenau laboratory research focus is to uncover the mechanisms that drive relapse in pediatric tumors with the long term goal of identifying novel therapeutic drug targets for treatment of relapse disease. Using novel zebrafish models of pediatric sarcoma and leukemia that mimic human malignancy, we have undertaken studies to discover novel therapies by addition of drugs to the water and imaging tumor growth in live zebrafish.
Click Here – Linardic Lab
Durham, NC – Sarcomas are among the most difficult-to-treat cancers in pediatric oncology, with metastatic forms having the highest mortality. We have established genetically defined human cell-based models for the pediatric skeletal muscle cancer known as rhabdomyosarcoma. Current therapies are based on xenograft models in immunocompromised mice, using established patient-derived patient cell lines, but because of the genetic variability of these cell lines, a true understanding of the causative role of certain genetic changes (e.g. chromosomal translocations) in rhabdomyosarcoma formation is not understood. Specific goals of this research program include the identification of signaling pathways corrupted in rhabdomyosarcoma, with focus on the PAX3-FOXO1 mutation and its downstream effectors, and identification of new therapeutic targets for treatment of this childhood cancer.
Click Here – Mal's Lab
Buffalo, NY – The research program in Dr. Mal's laboratory is focused on broad but highly integrated areas of study. The epigenetic gene regulation program involves identifying the epigenetic changes and deciphering the mechanism as a potential target strategy for therapy. This program is aimed to identify the epigenetic mechanism regulating normal and perturb differentiation. Particularly, the research focuses on epigenetic modulation of gene expression regulating skeletal muscle differentiation during development and regeneration, as well as in muscle tumor rhabdomyosarcoma (RMS). The drug discovery program involves searching small molecule modulators that would activate the skeletal muscle differentiation program in multipotent muscle stem cells (satellite cells) and in RMS tumor cells. Both programs are aimed at therapeutic applications for skeletal muscle associated tumors and degenerative diseases.
Click Here – Malkin Lab
Toronto, ON, Canada – Dr. Malkin's research interests are closely integrated with his clinical field of expertise. Specifically, his research program focuses primarily on genetic mechanisms of childhood cancer susceptibility, and the genetic basis of childhood sarcomas (cancers of bone, muscle and other soft tissues). His research team was the first to demonstrate that highly variable regions of DNA, termed copy number variations, are found in excess in the blood of some people, both children and adults, at very high risk of developing cancer, and may represent the earliest genetic changes that ultimately lead to development of cancer. Recently, his work has focused on application of this genetic/genomic information to develop rational clinical surveillance and treatment guidelines for children and adults deemed at genetic ‘high risk’ for cancer. In his sarcoma work, Dr. Malkin has studied the molecular and cell biology pathways that are associated with the development and progression of these cancers, and has identified molecules that might represent viable targets for novel drug therapies.
Rossella Rota Lab – Website in Progress
Rome, Italy – Our research interest is to investigate the mechanisms responsible for the inability of tumor cells to differentiate towards the cell lineage of origin. The main goal of our investigations on rhabdomyosarcoma, a soft-tissue sarcoma of myogenic origin, is the reactivation of the de-regulated pathways inhibiting skeletal muscle cell determination to achieve tumor cell cycle arrest and differentiation. Specifically, we evaluate, in in vitro and in vivo rhabdomyosarcoma models, the crosstalk among some developmental networks driving embryonic myogenesis such as transcription factors, chromatin modifying regulators and non-coding RNA to identify new therapeutic anti-cancer targets.
Click Here – Rudnicki Lab
Ottawa, ON, Canada – The Michael Rudnicki laboratory works to understand the molecular mechanisms that regulate the determination, proliferation, and differentiation of stem cells during embryonic development and during adult tissue regeneration. Located at the Sprott Centre for Stem Cell Research within the Regenerative Medicine Program at the Ottawa Hospital Research Institute, the lab has conducted extensive studies into both embryonic myogenesis and the function of myogenic satellite cells in adult skeletal muscle. Towards this end, the lab employs molecular genetic, genomic and proteomic approaches to determine the function and roles played by regulatory factors in stem cell function.
Click Here> – Schäfer Lab
Zurich, Switzerland – The main field of our research activity is the elucidation of the molecular mechanisms underlying development of pediatric sarcomas with special emphasis on the role of oncogenic fusion proteins generated by chromosomal translocations. Novel insights into these mechanisms are used in translational approaches to advance diagnosis and treatment in pediatric patients, carried out in close collaboration with clinical studies.
Click Here – Shipley Lab
London, UK – The current aims of the Sarcoma Molecular Biology Team are to identify therapeutic targets and/or molecular markers that will aid the treatment of patients with specific types of soft tissue sarcomas, as well as continuing a more minor interest in testicular germ cell tumours. Gene products relevant to sarcoma biology are identified through analyses of data from various screening approaches, including gene expression profiling, high-throughput sequencing and analysis of genomic copy number changes. This work is supported through use of extensive, well-characterised tumour collections. The functional relevance of selected gene products in specific sarcomas is evaluated using in vitro and in vivo experimental models.
Click Here – The Tapscott Lab
Seattle, WA – The Tapscott lab focuses on gene transcription in a chromatin context in normal development and disease. The lab uses the myogenic transcription factor MyoD to study how complex programs of gene expression unfold during cell differentiation. In addition, the lab studies gene expression in rhabdomysarcomas (cancers with characteristics of skeletal muscle) and human muscular dystrophies. Other areas of research in the lab include the formation of palindromes in the human cancer genome, gene and cell therapies for muscular dystrophy, and the biology of triplet repeats and their associated diseases.
Click Here – Triche Lab
Los Angeles, CA – The overarching goal of our research is to improve the outcome for patients with cancer. To do so, we seek to better understand the underlying genetic mechanisms that drive the development of cancer, or oncogenesis. In addition, we hope to utilize this newfound knowledge to identify therapeutic targets and deliver targeted therapies to tumor cells using nanoparticle delivery therapy.
Click Here – Zammit Lab
London, UK – The Zammit group's core research aims to understanding the regulation of satellite cell function in both normal and diseased muscle. The functional unit of skeletal muscle is the myofibre: a giant syncytial cell maintained by hundreds of myonuclei. Growth, maintenance and repair of the post-mitotic myofibres is performed by satellite cells. These resident stem cells are located on the edge of muscle fibres, below the surrounding basal lamina.
Click Here – Wager Lab
Cambridge, MA – Effective functioning of the body’s tissues and organs depends upon innate regenerative processes that maintain proper cell numbers (homeostasis) and replace damaged cells after injury (repair). In many tissues, regenerative potential is determined by the presence and functionality of a dedicated population of stem and progenitor cells, which respond to exogenous cues to produce replacement cells when needed. Understanding how these unspecialized precursors are maintained and regulated is essential for understanding the fundamental biology of tissues. In addition, this knowledge has practical implications, as the regenerative potential of tissue-specific stem and progenitor cells can be exploited therapeutically by transplantation (to replenish the stem cell pool) or by endogenous manipulation (to boost the repair activity of cells already present in the tissue).