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Gene Editing Pioneers Receive Americas Most Distinguished …

Posted: December 8, 2017 at 12:42 am

Gene Editing Pioneers Selected to Receive Americas Most Distinguished Prize in Medicine

August 15, 2017 – Albany, NY

For their roles in the creation of a remarkable gene editing system that has been called the discovery of the century, five researchers have been announced as the recipients of the Albany Medical Center Prize in Medicine and Biomedical Research for 2017. All five awardees have made important contributions to the development of CRISPR-Cas9, a gene engineering technology that harnesses a naturally occurring bacterial immune system process. The technology has revolutionized biomedical research and provided new hope for the treatment of genetic diseases and more. The awardees are:

The $500,000 award has been given annually since 2001 to those who have altered the course of medical research and is one of the largest prizes in medicine and science in the United States. It will be awarded on Wednesday, Sept. 27 during a celebration in Albany, New York.

The five recipients were chosen to receive the 2017 Albany Prize for their fundamental and complementary accomplishments related to CRISPR-Cas9. CRISPR is an acronym for clustered regularly interspaced short palindromic repeats, a DNA sequence found in the immune system of simple bacterial organisms.

The discovery of these CRISPR sequences in bacteria in the laboratory was the key to the later development of gene editing technology called CRISPR-Cas9 that has allowed scientists to easily and efficiently edit genes by splicing out and replacing or altering sections of DNA in the cells of any organism, including humans (though most current research uses isolated human cells in labs and animal models only). The editing technique has been compared to cutting and pasting words in a computer program.

CRISPR-Cas9 has revolutionized biological research in tens of thousands of laboratories worldwide. Its potential future applications include the possible ability to cure genetic defects such as muscular dystrophy, eradicate cancer, and allow for pig organs to safely be transplanted into humans. Its uses are so varied that CRISPR is being used to alter butterfly wing patterns and it could also someday help make crops hardier.

Though it cannot be used as a drug in patients yet, it is making a significant impact in the clinical world by accelerating drug research. Additionally, in laboratory experiments, CRISPR-Cas9 is being used to try to modify genes to block the HIV virus, and to attempt to change the DNA of mosquitos that carry the Zika virus so that it cannot be passed to humans.

Rarely has such a recent discovery transformed an entire field of research, as CRISPR has in biological research. Its implications for biological processes, including human health and disease are promising and quite profound, said Vincent Verdile, M.D. 84, the Lynne and Mark Groban, M.D. 69, Distinguished Dean of Albany Medical College and chair of the Albany Prize National Selection Committee. The Albany Prize recognizes that such a significant development in science is brought forth by a community of scientists, and, therefore, we felt it was appropriate to name a larger number of recipients than in the past.

CRISPR is an example of how science in the 21st century often works; as a remarkable ensemble act, in which a cast comes together to produce something that not one of them could do alone.

While most studies focus on gene editing in somatic (non-germline) cells, due to the profound ethical implications of modifying genes and impacting our species and environment, many CRISPR scientists, government representatives, ethicists and the general public are actively debating how we as a society ethically use the technology.

According to Dr. Verdile, the CRISPR story is a testament to the importance of basic biomedical research as the gateway to medical and scientific breakthroughs. The discovery of the CRISPR defense mechanism inside bacteria by basic scientists directly led to the development of the CRISPR gene editing system, which has promise for the treatment of disease.

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2017 Albany Prize Recipients

Emmanuelle Charpentier, Ph.D. Director, Department of Regulation in Infection Biology, Max Planck Institute for Infection Biology, BerlinVisiting Professor, Ume University, Sweden and Honorary Professor, Humboldt University

With her recent groundbreaking findings in the field of RNA-mediated regulation based on the CRISPR-Cas9 system, Dr. Charpentier laid the foundation for the development of the novel, highly versatile and precise genome engineering technology that has revolutionized life sciences research and opens new opportunities for the treatment of genetic disease.

She is co-inventor and co-owner of the fundamental intellectual property comprising the CRISPR-Cas9 technology, and co-founder of CRISPR Therapeutics and ERS Genomics, two companies that are developing the CRISPR-Cas9 genome engineering technology for biotechnological and biomedical applications.

Dr. Charpentier studied biochemistry, microbiology and genetics at the University Pierre and Marie Curie in Paris, and obtained her Ph.D. in microbiology for research performed at the Pasteur Institute in Paris. She continued her work at The Rockefeller University, New York University Langone Medical Center and the Skirball Institute of Biomolecular Medicine, all in New York City, and at St. Jude Childrens Research Hospital in Memphis.

She returned to Europe to establish a research group at the University of Vienna in Austria as assistant and associate professor. She was then appointed associate professor at the Laboratory for Molecular Infection Medicine Sweden at Ume University in Sweden where she is still a visiting professor.

In 2013, she was awarded an Alexander von Humboldt Professorship. She served as the head of the Department of Regulation in Infection Biology at the Helmholtz Centre for Infection Research in Braunschweig and professor at the Medical School of Hannover, Germany. In 2015, she was appointed scientific member of the Max Planck Society and director at the Max Planck Institute for Infection Biology in Berlin.

Jennifer Doudna, Ph.D. Professor, Molecular and Cell Biology and Chemistry, University of California, Berkeley

As an internationally renowned professor of chemistry and molecular and cell biology at U.C. Berkeley, Dr. Doudna and her colleagues rocked the research world in 2012 by describing a simple way of editing the DNA of any organism using an RNA-guided protein found in bacteria. This technology, called CRISPR-Cas9, has opened the floodgates of possibility for human and non-human applications of gene editing, including assisting researchers in the fight against HIV, sickle cell disease and muscular dystrophy.

Dr. Doudna is an investigator with the Howard Hughes Medical Institute and a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors and the American Academy of Arts and Sciences. She is also a Foreign Member of the Royal Society, and has received many other honors including the Breakthrough Prize in Life Sciences, the Heineken Prize, the BBVA Foundation Frontiers of Knowledge Award and the Japan Prize.

Dr. Doudna received her Ph.D. from Harvard University and was a postdoctoral research fellow in molecular biology at Harvard Medical School, Massachusetts General Hospital. She was the Lucille P. Markey Scholar in Biomedical Science at the University of Colorado. She later served on the faculty at Yale University as the Henry Ford II Professor of Molecular Biophysics and Biochemistry.

She is the co-author with Sam Sternberg of A Crack in Creation, a personal account of her research and the societal and ethical implications of gene editing.

Luciano A. Marraffini, Ph.D. Associate Professor, Laboratory of Bacteriology, The Rockefeller University, New York City

Dr. Marraffini made the seminal discovery that CRISPR-Cas works by cleaving DNA and was the first to propose that this system could be used for genome editing in heterologous systems. He then collaborated with Feng Zhang to perform the first successful editing experiment in eukaryotic (human) cells using CRISPR-Cas9. He continues to elucidate the molecular mechanisms of the CRISPR-Cas immune response in bacteria, including how sequences of viral and plasmid origin are selected to be integrated into CRISPR arrays and how different CRISPR-Cas systems found in different strains of bacteria attack their targets.Dr. Marraffini received his undergraduate degree from the University of Rosario in Argentina and his Ph.D. from the University of Chicago. He was a postdoctoral fellow at Northwestern University in the laboratory of Erik Sontheimer, after which he joined The Rockefeller University as assistant professor and the head of the Laboratory of Bacteriology in 2010. He was named a Howard Hughes Medical Institute-Simons Faculty Scholar in 2016. He is a recipient of the 2015 Hans Sigrist Prize and was named a finalist in the life sciences by the 2015 Blavatnik National Awards for Young Scientists. In 2014, Cell named him one of its 40 Under 40. He is a 2012 Rita Allen Foundation Scholar and a 2011 Searle Scholar, and is the recipient of an NIH Directors New Innovator Award and an RNA Society Award.

Francisco J.M. Mojica, Ph.D.Associate Professor of Microbiology, Department of Physiology, Genetics and Microbiology, University of Alicante, SpainMember of the Multidisciplinary Institute for the Study of the Environment Ramn Margalef, Spain

Dr. Mojicas pioneering work on CRISPR and his fundamental contribution to the knowledge of these components of bacteria for more than two decades makes him a leading scholar on the subject. Thanks to the contributions of Dr. Mojica in this field, exceptional laboratory tools, known as CRISPR-Cas technology, have been developed that can be used for the genetic manipulation of any living being, including humans. This technology has greatly simplified research in biology and medicine, for example, to study complex genetic processes such as those involved in embryonic development, carcinogenesis or neurodegenerative disorders. It is postulated that CRISPR-Cas technology will allow, in the near future, to cure diseases that are not curable or very difficult to tackle.

He received his Ph.D. in Biotechnology from the University of Alicante. He later completed two postdoctoral fellowships in laboratories at the University of Utah, Salt Lake City, and Oxford University in Great Britain. In 1997, he became professor of microbiology at the University of Alicante, founding the research group in molecular microbiology to resume the study on CRISPR he had initiated during his Ph.D. thesis work. In the last few years, his investigation has focused on the CRISPR immunization process, to understand how bacteria acquire foreign genetic material that make them resistant to infecting agents.

He has received many honors including the Lilly Foundation Award for Preclinical Biomedical Research, the Rey Jaime I Prize for Basic Research, and the BBVA Foundation Frontiers of Knowledge Award (biomedicine category).Feng Zhang, Ph.D.Core Member, Broad Institute of MIT and HarvardInvestigator at the McGovern Institute for Brain Research at MITThe James and Patricia Poitras Professor in Neuroscience and Associate Professor, Departments of Brain and Cognitive Sciences and Biological Engineering, Massachusetts Institute of Technology, Cambridge, Mass.

Dr. Zhang is a bioengineer developing and applying novel molecular technologies for studying the molecular and genetic basis of diseases and providing treatment. He played a seminal role in developing optogenetics, a powerful technology for dissecting neural circuits using light. Since joining the Broad and McGovern institutes in January 2011, Zhang has pioneered the development of genome editing tools for use in eukaryotic cells including human cells from natural microbial CRISPR systems.

Following his landmark demonstration that CRISPR-Cas9 could be harnessed for mammalian genome editing, his lab has continued to explore the CRISPR system and develop novel technologies for perturbing and editing the genome for disease research. He and his colleagues have successfully harnessed two additional CRISPR systems: CRISPR-Cpf1, which may allow simpler and more precise genome engineering, and CRISPR-Cas13a, a novel RNA-targeting system, which his team has adapted for use in rapid diagnostics.

Zhang leverages CRISPR and other methods to study the genetics and epigenetics of human diseases, especially complex disorders, such as psychiatric and neurological diseases, that are caused by multiple genetic and environmental risk factors and which are difficult to model using conventional methods. His labs tools, which he has made widely available, are also being used in the fields of immunology, clinical medicine, and cancer biology, among others. His long-term goal is to develop novel therapeutic strategies for disease treatment.He received his A.B. in chemistry and physics from Harvard College and his Ph.D. in chemistry from Stanford University.

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The Albany Medical Center Prize was established in 2000 by the late Morris Marty Silverman, a New York City businessman and philanthropist who grew up in Troy, N.Y., to honor scientists whose work has demonstrated significant outcomes that offer medical value of national or international importance. A $50 million gift commitment from the Marty and Dorothy Silverman Foundation provides for the prize to be awarded annually for 100 years.

Three previous Nobel Prize winners have been among the ranks of researchers honored, and five Albany Prize recipients have gone on to win the Nobel Prize, including Shinya Yamanaka, M.D., Ph.D., a leading stem cell scientist; Elizabeth Blackburn, Ph.D., who discovered the molecular nature of telomeres; Bruce Beutler, M.D., and the late Ralph Steinman, M.D., for their discoveries regarding the detailed workings of the immune system; and Robert Lefkowitz, M.D., for his work on cell receptors.

For biographies and downloadable photos of the 2017 recipients, and more information on the Albany Medical Center Prize in Medicine and Biomedical Research, visit: http://www.amc.edu/Academic/AlbanyPrize.

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