Conference Program. micrornas and their regulatory effects David P. Bartel, MIT Whitehead Institute for Biomedical Research, Cambridge, MA

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Conference Program Friday, December 4 5:30 p.m.-7:30 p.m. Plenary Session 1: Noncoding RNA Functions Session Chairperson: Joshua Mendell, UT Southwestern Medical Center, Dallas, TX Divergent transcription and long noncoding RNA in cancer Phillip A. Sharp, The David H. Koch Institute for Integrative Cancer Research at MIT, Cambridge, MA Genome regulation by long noncoding RNAs Howard Y. Chang, Stanford University, Stanford, CA micrornas and their regulatory effects David P. Bartel, MIT Whitehead Institute for Biomedical Research, Cambridge, MA Xist RNA in health and disease Jeannie T. Lee, HHMI/Massachusetts General Hospital, Boston, MA 7:30 p.m.-9:00 p.m. Opening Reception Saturday, December 5 8:00 a.m.-10:00 a.m. Plenary Session 2: Mechanisms of Noncoding RNAs in Tumorigenesis I Session Chairperson: David P. Bartel, MIT Whitehead Institute for Biomedical Research, Cambridge, MA Noncoding RNAs in cancer and development Andrea Ventura, Memorial Sloan Kettering Cancer Center, New York, NY Disregulation of lncrnas helps drive Ewing sarcoma pathogenesis Alejandro Sweet-Cordero, Stanford University, Stanford, CA mir-34 mirnas mediate the tumor suppressor functions of p53 Lin He, University of California, Berkeley, CA Strand-specific in vivo screen of cancer-associated mirnas unveils key drivers and oncogenic targets* Yejing Ge, The Rockefeller University, New York, NY The diverse roles of long noncoding RNAs in the p53 tumor suppressor pathway* Nadya Dimitrova, Yale University, New Haven, CT

10:00 a.m.-10:30 a.m. Break 10:30 a.m.-12:30 p.m. Plenary Session 3: Regulation of Noncoding RNAs in Cancer Session Chairperson: Howard Y. Chang, Stanford University, Stanford, CA Functional long noncoding RNAs in lung cancer Sven Diederichs, German Cancer Research Center, Heidelberg, Germany microrna biogenesis pathways in cancer Richard I. Gregory, Boston Children s Hospital, Boston, MA micrornas and lncrnas in mammalian physiology and cancer Joshua Mendell, UT Southwestern Medical Center, Dallas, TX A lncrna regulates DNA repair by homologous recombination* Vivek Sharma, National Cancer Institute, Bethesda, MD The role of long noncoding RNA HIF1A-AS2 in hypoxic environment of glioblastoma* Agnieszka Bronisz, Brigham and Women s Hospital/Harvard Medical School, Boston, MA 12:30 p.m.-2:30 p.m. Lunch on Own 2:30 p.m.-4:30 p.m. Plenary Session 4: Genomics of Noncoding RNAs in Cancer Session Chairperson: Anastasia Khvorova, University of Massachusetts Medical School, Worcester, MA Dependency of prostate cancer on HNRNPL and its associated RNAs X. Shirley Liu, Dana-Farber Cancer Institute, Boston, MA Mechanisms of tumorigenesis due to somatic mutations in DICER1 and DROSHA in childhood kidney cancers James F. Amatruda, UT Southwestern Medical Center, Dallas, TX It takes two to tango: The PVT1-MYC alliance in human cancer Anindya Bagchi, University of Minnesota Masonic Cancer Center, Minneapolis, MN Dicer1 deletion: Sufficient for angiosarcoma development and a haploinsufficient tumor suppressor in rhabdomyosarcoma* Mark Hatley, St. Jude Children s Research Hospital, Memphis, TN The enhancer landscape involves a core noncoding RNA protein interaction network for c-myc expression* Martin Walsh, Icahn School of Medicine at Mount Sinai, New York, NY 4:30 p.m.-5:00 p.m. Break

5:00 p.m.-6:00 p.m. Keynote Session Session Chairperson: Phillip A. Sharp, The David H. Koch Institute for Integrative Cancer Research at MIT, Cambridge, MA Noncoding RNAs of viral and cellular origin: Links to oncogenesis Joan Steitz, Yale University School of Medicine, New Haven, CT 6:00 p.m.-8:00 p.m. Poster Session A and Reception Sunday, December 6 8:00 a.m.-10:00 a.m. Plenary Session 5: Mechanisms of Noncoding RNAs in Tumorigenesis II Session Chairperson: Andrea Ventura, Memorial Sloan Kettering Cancer Center, New York, NY Regulatory RNAs Nikolaus Rajewsky, Max Delbrück Center for Molecular Medicine, Berlin, Germany Identification of trna-derived fragments that suppress cancer progression through displacement of transcripts from the YBX1 RNA-binding protein Sohail Tavazoie, The Rockefeller University, New York, NY In vivo analysis of linear and circular noncoding RNAs in tumorigenesis Pier Paolo Pandolfi, Beth Israel Deaconess Medical Center, Boston, MA Precise let-7 expression levels balance organ regeneration against tumor suppression* Liem Nyugen, UT Southwestern Medical Center, Dallas, TX LncRNA and microrna synergize to regulate colon cancer initiating cell asymmetric division* Xiling Shen, Duke University, Durham, NC 10:00 a.m.-10:30 a.m. Break

10:30 a.m.-12:00 p.m. Plenary Session 6: Biomarkers and Extracellular RNAs Session Chairperson: Sven Diederichs, German Cancer Research Center, Heidelberg, Germany The emergence of long noncoding RNAs in cancer Arul M. Chinnaiyan, University of Michigan, Ann Arbor, MI Illuminating the dark matter of the genome Michael T. McManus, University of California, San Francisco, CA Sequence-based design of small molecules targeting precursor micrornas Matthew D. Disney, The Scripps Research Institute Florida, Jupiter, FL 12:00 p.m.-3:00 p.m. Poster Session B and Lunch 3:00 p.m.-4:30 p.m. Plenary Session 6 (continued): Biomarkers and Extracellular RNAs Session Chairperson: X. Shirley Liu, Dana-Farber Cancer Institute, Boston, MA Isolation of extracellular nucleic acid and their diagnostic and biomarker potential Thomas Tuschl, The Rockefeller University, New York, NY Cancer cell extracellular vesicles trigger apoptosis specifically in a primary cells Thomas R. Gingeras, Cold Spring Harbor Laboratory, Cold Spring Harbor, NY microrna-24 transferred from platelet-derived microparticles to tumor cells in solid tumors targets mt-nd2 mrna and modulates mitochondrial function and tumor growth* James Michael, Temple University School of Medicine, Philadelphia, PA HiClinc-1, a highly conserved Cancer-Testis lncrna, regulates cell proliferation and tumor onset* Yasuyuki Hosono, University of Michigan Medical School, Ann Arbor, MI 4:30 p.m. - 5:30 p.m. Panel Discussion on Competitive Endogenous RNAs Moderator: Howard Y. Chang, Stanford University, Stanford, CA Panelists: Pier Paolo Pandolfi, Beth Israel Deaconess Medical Center, Boston, MA David Bartel, Massachusetts Institute of Technology, Cambridge, MA Phillip A. Sharp, The David H. Koch Institute for Integrative Cancer Research at MIT, Cambridge, MA 5:00 p.m.-5:30 p.m. Discussion 5:30 p.m.- Evening Off / Dinner on Own

Monday, December 7 8:00 a.m.-10:00 a.m. Plenary Session 7: Science of RNA Therapeutics and Delivery Session Chairperson: Jeannie T. Lee, HHMI/Massachusetts General Hospital, Boston, MA Targeting nuclear noncoding RNAs David R. Corey, UT Southwestern Medical Center, Dallas, TX microrna-based therapeutics in cancer Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, Boston, MA Regulatory control of lincrna function through formation of complex RNA structural motifs Anna Pyle, Yale University, New Haven, CT Differentiation of mammary tumors and reduction in metastasis upon Malat1 lncrna loss* Gayatri Arun, Cold Spring Harbor Laboratory, Cold Spring Harbor, NY Robust modulation of gene expression in aggressive glioblastoma mouse models: A new approach for in vivo target validation* Andrew Coles, University of Massachusetts Medical School, Worcester, MA 10:00 a.m.-10:30 a.m. Break 10:30 a.m.-12:30 p.m. Plenary Session 8: Clinical Translation of Noncoding RNA Therapies Session Chairperson: Frank J. Slack, BIDMC Cancer Center/Harvard Medical School, Boston, MA Selective activation of gene expression by targeting long noncoding RNA James Barsoum, RaNA Therapeutics, Cambridge, MA Advances in oligonucleotide chemistry for the treatment of neurodegenerative disorders and brain tumors Anastasia Khvorova, University of Massachusetts Medical School, Worcester, MA Towards a therapy for Angelman syndrome by targeting a long noncoding RNA to active UBE3A Frank Rigo, Isis Pharmaceuticals, Carlsbad, CA Development of a microrna mimic delivered by targeted nanocells as a treatment for patients with recurrent thoracic cancer* Glen Reid, Asbestos Diseases Research Institute, Sydney, Australia Potent knock down of lncrnas in vitro and in vivo with antisense LNA GapmeRs* Frandsen Niels, Exiqon, Vedbaek, Denmark 12:30 p.m.-12:45 p.m. Closing Remarks