Gene-Editing Pioneers to Receive $500K Prize for the Development of CRISPR-Cas9

Scientists’ contributions “revolutionized biomedical research”

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:

  • Emmanuelle Charpentier, PhD, Director of the Department of Regulation in Infection Biology at the Max Planck Institute for Infection Biology in Germany, and Honorary Professor at Humboldt University and Visiting Professor at Umeå University in Sweden
  • Jennifer Doudna, PhD, Professor of Molecular and Cell Biology and Chemistry at the University of California, Berkeley
  • Luciano Marraffini, PhD, Associate Professor in the Laboratory of Bacteriology at The Rockefeller University in New York City
  • Francisco J.M. Mojica, PhD, Associate Professor of Microbiology in the Department of Physiology, Genetics, and Microbiology at University of Alicante and a member of the Multidisciplinary Institute for the Study of the Environment Ramón Margalef, both in Spain
  • Feng Zhang, PhD, Core Member at the Broad Institute of MIT and Harvard, and the James and Patricia Poitras Professor in Neuroscience and Associate Professor in the Departments of Brain and Cognitive Sciences and Biological Engineering at Massachusetts Institute of Technology in Cambridge, Massachusetts.

 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, September 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. In addition, 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 mosquitoes 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, MD, the Lynne and Mark Groban 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 (nongermline) 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.

Source: Albany Medical Center; August 15, 2017.