Getting the message: How elucidation of messenger RNA formation empowered RNA therapeutics

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2021 Warren Alpert Foundation Prize Virtual Symposium

In honor of Lynne Maquat and Joan Steitz for the discovery of fundamental pathways and mechanisms that ensure accurate RNA splicing and quality control of gene expression involving RNA.

Lynne Maquat

Lynne Maquat | 2021 Recipient

Dr. Lynne Maquat is the J. Lowell Orbison Endowed Chair, Professor of Biochemistry & Biophysics who holds concomitant appointments in Pediatrics and in Oncology, Founding Director of the Center for RNA Biology, and Founding Chair of Graduate Women in Science at the University of Rochester, Rochester, NY. After obtaining her PhD in Biochemistry from the University of Wisconsin-Madison and undertaking post-doctoral work at the McArdle Laboratory for Cancer Research, she joined Roswell Park Cancer Institute before moving to the University of Rochester. Dr. Maquat’s research focuses on the molecular basis of human diseases, with particular interest in mechanisms of mRNA decay. Dr. Maquat discovered nonsense-mediated mRNA decay (NMD) in human diseases in 1981 and, subsequently, the exon-junction complex (EJC) and how the EJC marks mRNAs for a quality-control “pioneer” round of protein synthesis. She also discovered Staufen-mediated mRNA decay, which mechanistically competes with NMD and, by so doing, new roles for short interspersed elements and long non-coding RNAs. Additionally, she has defined a new mechanism by which microRNAs are degraded, thereby regulating mRNAs so as to promote the cell cycle.

Maquat is an elected Fellow of the American Association for the Advancement of Science (2006); an elected Member of the American Academy of Arts & Sciences (2006), the National Academy of Sciences (2011), and the National Academy of Medicine (2017); and a Batsheva de Rothschild Fellow of the Israel Academy of Sciences & Humanities (2012-3). She received the William C. Rose Award from the American Society for Biochemistry & Molecular Biology (2014), a Canada Gairdner International Award (2015), the international RNA Society Lifetime Achievement Award in Service (2010) and in Science (2017), the FASEB Excellence in Science Award (2018), the Vanderbilt Prize in Biomedical Science (2017), the Wiley Prize in Biomedical Sciences (2018), the International Union of Biochemistry and Molecular Biology Medal (2019), and the Wolf Prize in Medicine (2021). Maquat is well-known for her efforts to promote women in science.

Joan Steitz

Joan Steitz | 2021 Recipient

Joan A. Steitz, Ph.D., is the Sterling Professor of Molecular Biophysics and Biochemistry at Yale University School of Medicine and a Howard Hughes Medical Institute Investigator.Steitz earned her BS in chemistry from Antioch College in 1963. Significant findings from her work emerged as early as 1967, when her Harvard PhD thesis with Jim Watson examined the test-tube assembly of a ribonucleic acid (RNA) bacteriophage (antibacterial virus) known as R17.

Steitz spent the next three years in postdoctoral studies at the Medical Research Council Laboratory of Molecular Biology in Cambridge, England, where she used early methods for determining the biochemical sequence of RNA to study how ribosomes know where to initiate protein synthesis on bacterial mRNAs. In 1970, she was appointed assistant professor of Molecular Biophysics and Biochemistry at Yale, becoming full professor in 1978. At Yale, she established a laboratory dedicated to the study of RNA structure and function. In 1979, Steitz and her colleagues described a group of cellular particles called small nuclear ribonucleoproteins (snRNPs), a breakthrough in understanding how RNA is spliced. Subsequently, her laboratory has defined the structures and functions of other noncoding RNPs, such as those that guide the modification of ribosomal RNAs, microRNAs and several produced by transforming herpesviruses.

Steitz is an investigator of the Howard Hughes Medical Institute, a member of the American Academy of Arts and Sciences, American Philosophical Society, National Academy of Sciences, and Institute of Medicine. Her many honors include the U.S. Steel Foundation Award in Molecular Biology (1982); National Medal of Science (1986); FASEB Excellence in Science Award (2003); RNA Society Lifetime Achievement Award (2004); Gairdner Foundation International Award (2006); Albany Medical Center Prize in Medicine and Biomedical Research (2008) [shared with Elizabeth Blackburn]; Pearl Meister Greengard Prize (2012); La grande médaille 2013 de l'Académie des sciences, Insititut de France; Foreign Member of the Royal Society of London (2014); Herbert Tabor Award, American Society of Biochemistry and Molecular Biology (2015); Biopolymers Murray Goodman Memorial Prize, American Chemical Society (2015); William Clyde DeVane Award for Teaching Excellence, Yale University (2016); Jonathan Kraft Prize for Excellence in Cancer Research (2016); ASCB Inaugural Fellow (2016); Lasker~Koshland Special Achievement Award in Medical Science, the Albert and Mary Lasker Foundation  (2018); and the Wolf Prize in Medicine (shared with Lynne Maquat and Adrian Krainer). Dr. Steitz has been awarded 19 honorary degrees.

Symposium Program

Each year the recipient(s) of the Warren Alpert Foundation Prize are recognized at a scientific symposium hosted by Harvard Medical School.

Opening Remarks

George Q. Daley, MD, PhD

Dean of Harvard Medical School; Caroline Shields Walker Professor of Medicine

Moderator

Karen Adelman, PhD

Professor of Biological Chemistry and Molecular Pharmacology; Harvard Medical School

Presentations

Phillip Sharp, PhD

Institute Professor, Koch Institute and Department of Biology Massachusetts Institute of Technology

RNA Condensates in Transcription and RNA Splicing

Joan Steitz, PhD

Sterling Professor of Molecular Biophysics and Biochemistry; Investigator, Howard Hughes Medical Institute Yale University School of Medicine

Viral Noncoding RNAs: New Functions, New Structures

Lynne E. Maquat, PhD

J. Lowell Orbison Endowed Chair, Professor of Biochemistry & Biophysics University of Rochester Medical Center

Nonsense-mediated mRNA Decay and Human Disease: Guardian and Executor of Gene Expression

Eugene Yeo, PhD

Professor of Cellular & Molecular Medicine University of California San Diego

RNA binding proteins as regulators, drugs and drug targets

Melissa J. Moore, PhD

Chief Scientific Officer, Platform Research Moderna Therapeutics

RNA as Medicine

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Past Symposia

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I was shocked and humbled that I would be considered for this prestigious award with four outstanding scientists, Francis Collins, Lap-Chee Tsui, Michael Welsh and Paul Negulescu. The Alpert Award is very meaningful to me, especially as an alumna of Harvard Medical School. Receiving the award recognizes the scientific and therapeutic revolution that has occurred over the past three decades in the field of cystic fibrosis. The award also acknowledges the importance of team science spanning from basic to clinical investigation that is required to translate our understanding of molecular biology into life-changing new therapies. I am honored to receive this award.
- Bonnie Ramsey

Bonnie Ramsey | 2018 Recipient

Bonnie Ramsey MD is Professor and Vice Chair for Research in the Department of Pediatrics and holds the Endowed Chair in Cystic Fibrosis at the University of Washington School of Medicine.  She is also the Director of the Center for Clinical and Translational Research (CCTR) at Seattle Children’s Research Institute and is the co-PI of the University of Washington Institute of Translational Health Sciences (ITHS) supported by the NCATS Clinical Translational Science Award (CTSA). She is an elected member of the National Academy of Medicine

Dr. Ramsey received her BA from Stanford University in 1972 and her MD from Harvard Medical School in 1976. After pediatric residency training at Boston Children’s Hospital, she moved Seattle Children’s in 1978 first as a resident and fellow and then became an attending physician in 1980. Her career has focused on clinical care and research in the field of Cystic Fibrosis (CF). She is internationally recognized for her work in developing new therapies for patients with CF. She is also interested in the ethics of pediatric clinical research and has served on two Institute of Medicine committees focused on this topic.

Her primary research interest has been better understanding Pseudomonas aeruginosa (Pa) infections in the CF airway in order to develop better therapies. Over the past 2 decades she has led a large natural history of early Pa infection in young children with CF, the Early Pa Infection Control(EPIC) study, creating one of the world’s largest data and microbiological specimen collection in this population. Since the 1990’s, she has studied the role of inhaled antibiotics in treatment of Pa infections including the development of inhaled tobramycin ( TOBI®) initially for patients with established infections and more recently for eradication of early infections.

For nearly the past two decades, Bonnie directed the Coordinating Center for the Cystic Fibrosis Foundation Therapeutics (CFFT) Development Network (TDN), a national clinical trials network that has successfully conducted therapeutic trials assisting in the development of novel treatments for patients with this disorder. Several of the drugs have reached FDA approval, significantly impacting the lives of patients with CF. She now serves as a Senior Consultant to the TDN.

 View Past Recipients