The CRISPR/Cas9 Revolution and Gene Editing

2016 Warren Alpert Foundation Prize Symposium

In honor of Rodolphe Barrangou, Emmanuelle Charpentier, Jennifer Doudna, Philippe Horvath, Viginijus Siksnys for remarkable contributions to the understanding of the CRISPR bacterial defense system and the revolutionary discovery that it can be adapted for genome editing.

Rodolphe Barrangou

Rodolphe Barrangou | 2016 Recipient

Rodolphe Barrangou is an Associate Professor in the Department of Food, Bioprocessing and Nutrition Sciences at North Carolina State University, a NC State University Scholar, and the Todd R. Klaenhammer Distinguished Scholar in Probiotics Research. Dr. Barrangou is also an associate member of the Microbiology graduate program, the Biotechnology graduate program, the Functional Genomics graduate program, and the Center for Integrative Medicine. Dr. Barrangou is also an adjunct member of the Food Science Department at the Pennsylvania State University.

His CRISPR laboratory focuses on the evolution and functions of CRISPR-Cas systems, and their use for bacterial genotyping, building prokaryotic immunity, and Cas9-mediated genome editing in lactic acid bacteria used in food manufacturing.

Dr. Barrangou earned a BS in Biological Sciences from the Rene Descartes University in Paris, France; a MS in Biological Engineering from the University of Technology in Compiegne, France; a MS in Food Science from NC State University; a PhD in Genomics from NC State University; and a MBA from the University of Wisconsin-Madison. Dr. Barrangou and colleagues at DuPont established the biological role of CRISPR-Cas systems in adaptive immunity in bacteria, and used CRISPR-based technologies for bacterial genotyping of industrial cultures, and for the vaccination of dairy cultures against bacteriophages. After nine years in R&D and M&A at Danisco and DuPont, he joined the faculty at NC State University in 2013.

Dr. Barrangou is the recipient of the 2014 NC State Alumni Association Outstanding Research Award, and of the 2015 NC State Faculty Scholars Award. He has been on the Thomson Reuters Highly Cited Researchers list in 2014 and 2015. Dr. Barrangou is on the board of directors of Caribou Biosciences, a co-founder and member of the Scientific Advisory Board of Intellia Therapeutics, and a founding investor of Locus Biosciences.

Dr. Barrangou has published numerous articles on CRISPR-Cas systems and their use since 2005, including establishing their role as bacterial immune systems, and exploiting them for industrial applications. Following the initial work unraveling the biological function of CRISPR arrays and cas genes, subsequent studies and collaborative efforts identified PAMs as critical sequences for phage DNA targeting, showed that Cas9 is an endonuclease which can cleave plasmid and phage DNA, and provided the first proof of concept that CRISPR can be reprogrammed and transferred heterologously. Dr. Barrangou also established and co-hosted five international CRISPR meetings, and edited the first book on CRISPR-Cas systems.

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”

Jennifer Doudna

Jennifer Doudna | 2016 Recipient

Jennifer Doudna is the Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences and she is Professor of Molecular and Cell Biology and Professor of Chemistry at UC Berkeley and an Investigator of the Howard Hughes Medical Institute.  Prof. Doudna’s research seeks to understand how RNA molecules control the expression of genetic information. Her research led to insights about CRISPR-Cas9-mediated bacterial immunity that enabled her lab and that of collaborator Emmanuelle Charpentier to re-design this system for efficient genome engineering in animals and plants, creating a transformative technology that is revolutionizing the fields of genetics, molecular biology and medicine. She is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, the National Academy of Medicine and the National Academy of Inventors. She is a recipient of awards including the NSF Waterman Award, the FNIH Lurie Prize, the Paul Janssen Award for Biomedical Research, the Breakthrough Prize in Life Sciences, the Princess of Asturias Award (Spain), the Gruber Prize in Genetics, the Massry Prize and the L’Oreal-UNESCO International Prize for Women in Science.

Philippe Horvath

Philippe Horvath | 2016 Recipient

Philippe Horvath is a senior scientist at DuPont.  He graduated from Université Louis-Pasteur, Strasbourg, France in 1996 and obtained his Ph.D. in Cellular and Molecular Biology in 2000. That same year, he was recruited by Rhodia Food and worked at the R&D center in Dangé-Saint-Romain, France, where he contributed to the development of molecular biology tools for bacterial strain screening, microbial identification, and typing of lactic acid bacteria and their bacteriophages.

Philippe became senior scientist in 2006, two years after Rhodia Food was acquired by the Danish company Danisco, a world leader in specialty food ingredients. In 2014, three years after DuPont acquired Danisco, Philippe was appointed Associate to the DuPont Fellows Forum, and further appointed DuPont Nutrition & Health Technical Fellow in 2015.

Since late 2002, a large part of Philippe’s research activities has been dedicated to CRISPR (clustered regularly interspaced short palindromic repeats), first as a polymorphic chromosomal region useful for strain differentiation and tracking, and then as a bacterial immune system with considerable industrial, biotechnological, and medical applications. Philippe is co-inventor of 95 patents and/or patent applications, of which 62 are related to various uses of CRISPR, and co-author of 31 peer-reviewed articles (22 about CRISPR) and 4 book chapters. Together with other scientists in the company, Philippe was recognized with the 2008 Danisco Innovation Award, and with the 2013 Bolton/Carothers Innovative Science Award. In addition to being selected as a 2015 Thomson Reuters Highly Cited Researcher, Philippe was awarded with the 2015 Massry Prize.

Virginijus Siksnys

Virginijus Siksnys | 2016 Recipient

Dr. Virginijus Siksnys studied chemistry at Vilnius University and obtained his Ph.D. from Moscow State University before returning to Vilnius where he moved through different research ranks at the Institute of Applied Enzymology/Institute of Biotechnology. He holds the position of Professor of Vilnius University since 2002 and is Chief scientist/Department head at the Institute of Biotechnology of Vilnius University. He also serves as a Chairman of the Institute’s Board. He is a member of Lithuania Academy of Sciences and his work has been recognized with several awards including the Lithuania National Science Prize. Dr Siksnys has made a major and sustained contribution to the understanding of the structure and function of restriction enzymes. His current research on the CRISPR system for antiviral defense has had a major impact on the field. His studies of the Cas9 protein paved the way for development of novel tools for genome editing applications.

Symposium Program

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

Featured Speakers include:

Rodolphe Barrangou, PhD

Todd R. Klaenhammer Distinguished Scholar in Probiotics Research North Carolina State University

CRISPR-mediated immunity in bacteria: discovery and applications

Austin Burt, PhD

Professor of Evolutionary Genetics Imperial College London

Developing CRISPR-based gene drive for malaria control

Emmanuelle Charpentier, PhD

Prof. Dr.; Scientific Member of the Max Planck Society, Max Planck Director Professor, Umeå University

The transformative genome engineering CRISPR-Cas9 technology: lessons learned from bacteria

Jennifer Doudna, PhD

Li Ka Shing Chancellor’s Chair in Biomedical and Health Sciences/HHMI Investigator University of California, Berkeley

The Future of Genome Engineering: Biology, Technology and Ethics

Philippe Horvath, PhD

Senior Scientist

Dupont CRISPR-mediated immunity in bacteria: discovery and applications

Virginijus Siksnys, PhD

Professor and Chief Scientist/Department Head, Institute of Biotechnology Vilnius University

From mechanisms of microbial immunity to novel genome editing tools

Luhan Yang, PhD

Chief Scientific Officer

eGenesis Rewriting the pig genome to transform Xenotransplantation

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I would like to express my sincere appreciation to the Warren Alpert Foundation and Harvard Medical School for awarding me such a great honor. For me, this award is not only an honor but a responsibility.
- Tu Youyou

Tu Youyou | 2015 Recipient

For their pioneering discoveries in chemistry and parasitology, and personal commitments to translate these into effective chemotherapeutic and vaccine-based approaches to control malaria - their collective work will impact millions of lives globally particularly in the developing countries.

Tu Youyou is currently a Professor and Director of Qinghaosu Research Center at Institute of Chinese Materia Medica, China Academy of Chinese Medical Sciences.

Tu joined the Institute of Chinese Materia Medica (ICMM), China Academy of Traditional Chinese Medicine (CATCM) (renamed China Academy of Chinese Medical Sciences, CACMS in 2005) in 1955 after graduation from School of Pharmacy, Beijing Medical College (1951 – 1955).  She late attended a two and half year course on the traditional Chinese medicine (TCM) (1959-1962) - an in depth training specifically designed and offered to the graduates with Western medical background.  Over more than forty years, Tu has been working in the institute as a research assistant, assistant professor, associate professor, professor and director of the Chemistry Department.  She also holds a chief professor position of CACMS as well as members of Phytochemistry Society of China Pharmaceutical Association; Chinese Society of Traditional and Natural Drugs; Council member and Founding Member of Chinese Association of Invention; Executive Committee of All-China Women's Federation (1988-93) and a committee member of Beijing Committee of Natural Science Foundation (1995-97).

Tu’s research on anti-malaria drugs started in 1969 when she was appointed to head the Project 523 research group at the institute.  Project 523 was a secret military program initiated by the Chinese leadership in 1967 in supporting Vietnam government for searching medicines to treat anti chloroquine-resistant malaria.  Tremendous efforts had been made both in US and China but no effective drugs were found by the time Tu accepted the task.

Tu started with collection of over 2000 candidate recipes from which she and her team later screened over hundreds of herbal extracts.  Only one of the extracts from Qinghao (Artemisia annua L.) showed some promising effect, which however was not consistent and reproducible.  Tu further reviewed all available tradition Chinese medicine literatures and noticed a paragraph “Take a handful of Qinghao, soak in two liters of water, strain the liquid, and drink” in the Handbook of Prescriptions for Emergencies (340 CE) authored by Ge Hong.  She realized that the heating applied during extraction might have damaged the active components in the herb.  With this in mind, Tu re-designed low temperature extraction approaches using the solvents with low boiling points.  On the 4th October 1971, a neutral ether extract numbered 191 was found 100% effective in clearing Plasmodia in the mice and monkey test models.  To prove safety of the Qinghao extract and expedite the project, Tu and other two colleagues volunteered in the toxicity study on themselves in July 1972.  Between August and October 1972, Tu and her team carried out the first clinical trial in southern China in which all thirty-one patients treated with the Qinghao extract recovered from the disease.  Subsequent to the first clinical trial, Tu and her colleagues further purified the extract and obtained a pure active crystalline in November 1972.  The compound was late named Qinghaosu (Artemisinin).

Over last several decades, Tu has continued her effort in fighting against malaria.  She and her colleagues developed artemisinin into a medicine and late the team further developed dihydro-artemisinin, another anti-malaria drug, which is ten fold more effective than artemisinin itself.

Tu’s achievement in the discovery of artemisinin and its application in the malaria treatment as well as her unanimous contribution to the healthcare of human being have been well recognized nationally and internationally.  She was honored the Lasker ~ Debakey Clinical Medical Research Award in 2011.  Tu also received numerous awards nationally which include “Award for Progress in Anti-malarial Research Achieved by Project 523 Scientific Team” honored by China National Congress of Science and Technology (1978); “National Scientific Discovery Award” for Anti-malaria Drug-Qinghaosu by the China Ministry of Science and Technology (1979); “Invention Award” (as the first inventor) by China National Congress for Awards in Science and Technology (1982); “Award of Young and Middle-aged Experts with Outstanding Contribution” by the Chinese Government (1984); “The Top Honorary Award” by China Academy of Traditional Chinese Medicine (1992); “The Top Ten National Achievements for Progress in Science and Technology” by China National Committee of Science and Technology (1992); “First-Class Award of National Achievements in Science and Technology” by China National Award Committee for Advances in Science and Technology (1992); “National Model” by China State Council (1995); “Award for Outstanding Achievement in Traditional Chinese Medicine” by Guangzhou Zhongjing Award Foundation for Traditional Chinese Medicine (1995); “Outstanding Scientific Achievement Award” by Qiu Shi Science and Technologies Foundation of Hong Kong (1996); “Top Ten Health Achievements in New China” by China Ministry of Health (1997); “Female Inventor of the New Century” by China National Bureau of Intellectual Property (2002); “Golden Medal of the 14th National Invention Exhibition” by China National Bureau of Intellectual Property (2003); “Award for Development of Chinese Materia Medica” by Cyrus Chung Ying Tang Foundation, (2009).  She also received “Prince Mahidol Award” by Thailand Prince Mahidol Award Foundation (2003),  “GlaxoSmithKline Award for Outstanding Achievements in Life Science” (2011), Lasker~DeBakey Clinical Medical Research Award(2011), and the Nobel Prize in Physiology or Medicine(2015).

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