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 am honored to receive this award in recognition of our seminal work published in Nature in 2011 and in Science in 2012, including the discovery of tracrRNA and the delineation of its key role in the targeting and editing of DNA by CRISPR-Cas9. This work is a wonderful example of the importance of basic research, demonstrating its relevance for translational science and important medical applications.
- Emmanuelle Charpentier

Emmanuelle Charpentier | 2016 Recipient

Emmanuelle Charpentier studied biochemistry, microbiology and genetics at the University Pierre and Marie Curie, Paris, France and obtained her PhD in Microbiology for her research performed at the Pasteur Institute. She then continued her work in the United States, at The Rockefeller University, New York University Langone Medical Center and the Skirball Institute of Biomolecular Medicine (all in New York, NY) and at St Jude Children's Research Hospital (in Memphis, TN). E. Charpentier returned to Europe to establish her own research group at the Max F. Perutz Laboratories of the University of Vienna in Austria where she habilitated in the field of Microbiology. She was then appointed Associate Professor at the Laboratory for Molecular Infection Medicine Sweden (MIMS, part of Nordic European Molecular Biology Laboratory (EMBL) Partnership for Molecular Medicine) at Umeå University in Sweden where she habilitated in the field of Medical Microbiology and is still active as a Visiting Professor. Between 2013 and 2015, E. Charpentier was Head of the Department of Regulation in Infection Biology at the Helmholtz Centre for Infection Research, Braunschweig, and Professor at the Medical School of Hannover in Germany. In 2013, she was awarded an Alexander von Humboldt Professorship, which she has held since 2014. In 2015, E. Charpentier was appointed Scientific Member of the Max Planck Society in Germany and Director at the Max Planck Institute for Infection Biology in Berlin, Germany.

E. Charpentier is recognized as a world-leading expert in regulatory mechanisms underlying processes of infection and immunity in bacterial pathogens. Her work has led to a number of seminal discoveries and insights into pathways governing antibiotic resistance and virulence of bacterial pathogens. With her recent groundbreaking findings in the field of RNA-mediated regulation based on the CRISPR-Cas9 system, E. Charpentier has laid the foundation for the development of a novel, highly versatile and specific genome editing technology that is revolutionizing life sciences research and could open up whole new opportunities in biomedical gene therapies. The resulting field of research is now developing at dazzling speed, with exciting new aspects emerging almost weekly. E. Charpentier is Elected Foreign Member of The Royal Swedish Academy of Sciences 2015, Elected Member German National Academy of Sciences 2015, Elected Member of the European Academy of Microbiology 2015, Elected Fellow of the American Academy of Microbiology in 2015 and Elected EMBO Member in 2014. E. Charpentier has been awarded prestigious honors including the Paul Ehrlich and Ludwig Darmstaedter Prize 2016, French Chevalier Order de la Légion d’Honneur in 2016, World Technology Award for Biotechnology 2015, Leibniz Prize 2016, a 2016 Vallee Visiting Professorship, the 2016 HFSP Nakasone Award, an Honorary Doctorate of the KU Leuven, the Science Award of Lower Saxony 2015, the ABRF Annual Award for Outstanding Contributions to Biomolecular Technologies 2016, the Massry Prize 2015, the Otto Warburg Medal 2016, the L’Oréal-UNESCO For Women in Science Award 2016, the Carus-Medal of the German National Academy of Sciences Leopoldina 2015, the Umeå University EC Jubilee Award in 2015, the Gruber Prize in Genetics 2015, the Princess of Asturias Award for Technical and Scientific Research 2015, the 11th International Society for Transgenic Technologies Prize, the Hansen Family Award 2015, the 2015 Louis Jeantet Prize for Medicine, the 2015 Ernst Jung Prize for Medicine, the 2015 Breakthrough Prize in Life Sciences, the 2014 Grand Prix Jean-Pierre LeCocq, the 2014 Jacob Heskel Gabbay Award in Biotechnology and Medicine, the 2014 Dr Paul Janssen Award, the 2014 Göran Gustafsson Prize, an Alexander von Humboldt Professorship in 2013 and the Eric K. Fernström Prize in 2011. The impact of her scientific accomplishments has also been recognized in the broader community of world affairs. E. Charpentier was selected as one of TIME’s 100 Most Influential People in the World in 2015, one of Foreign Policy’s 100 Leading Global Thinkers in 2014, one of Vanity Fair’s 50 most influential French people worldwide in 2014.

E. Charpentier is inventor and co-owner of seminal intellectual property comprising the CRISPR-Cas9 technology, and co-founder of CRISPR Therapeutics and ERS Genomics, created to facilitate the development of the CRISPR-Cas9 genome engineering technology for biotechnological and biomedical purposes.

(2016-03-06) picture: “Hallbauer&Fioretti”

 View Past Recipients