Jennifer Doudna, Ph.D. , professor, Molecular and Cell Biology and Chemistry, University of California, Berkeley. (UC-Berkeley)

Jennifer Doudna, Ph.D. , professor, Molecular and Cell Biology and Chemistry, University of California, Berkeley. (UC-Berkeley)

Luciano Marraffini, Ph.D., associate professor, Laboratory of Bacteriology, The Rockefeller University, New York City. (Mario Morgado)

Luciano Marraffini, Ph.D., associate professor, Laboratory of Bacteriology, The Rockefeller University, New York City. (Mario Morgado)

Gene-editing scientists to share $500K Albany Med prize

Albany

Five scientists whose work on the revolutionary gene-editing technology CRISPR will share the 2017 Albany Medical Center Prize in Medicine and Biomedical Research.

The decision by the Albany Prize National Selection Committee to award the $500,000 prize to these researchers stands out from recent announcements of the prestigious award, which have acknowledged scientists for groundbreaking work leading to current medical advances. While developments using CRISPR have exploded this year, its use in humans remains a promise, but one with far-reaching effects.

"The committee saw this technology as having huge potential for eradicating human disease," said Dr. Vincent Verdile, dean of Albany Medical College and chair of the prize committee.

CRISPR (pronounced "crisper") stands for "clustered regularly interspaced short palindromic repeats." It is a DNA sequence that simple bacteria use to defend themselves against viruses by snipping out part of the virus DNA so it can be recognized by the bacteria's own immune systems. The technology based on it lets scientists "edit" genes at specific locations by removing, adding or altering parts of the DNA sequence.

In the last year, CRISPR technology has been used to remove a gene linked to heart disease from human embryos and to create a cancer-killing gene that shrinks tumors in mice. Last week, scientists revealed in the journal Science that they had created piglets stripped of viruses that could cause disease in humans; the technique could open the door for eventual transplantation of livers, hearts and other organs from pigs to people.

The scientists who will share the Albany Prize are:

Emmanuelle Charpentier of the Max Planck Institute for Infection Biology in Germany. Charpentier is co-inventor and co-owner of the intellectual property comprising the CRISPR gene-editing system, and co-founder of two companies developing the technology for biotech and biomedical applications.

Jennifer Doudna of the University of California, Berkeley. Five years ago, Doudna described a simple way of editing the DNA of any organism using an RNA-guided protein founded in bacteria.

Luciano Marraffini of Rockefeller University in New York City. Marraffini discovered that CRISPR works by severing DNA and was the first to propose that it could be used to edit genes in organisms other than bacteria. With Feng Zhang, he performed the first successful CRISPR gene-editing experiment in human cells.

Francisco J.M. Mojica of the University of Alicante in Spain. Mojica's work has led to the development of tools used in the genetic manipulation of any living being, including humans.

Feng Zhang of the Broad Institute of Massachusetts Institute of Technology and Harvard University. Zhang pioneered the development of gene editing tools for use in human cells from bacterial CRISPR systems.

The Albany Prize Committee's selection of five scientists to share the award this year reflects an increasing trend in science toward collaboration, where information is shared and groups of researchers move knowledge forward in ways that no one of them could do alone, Verdile said. It's a major change since the days when a single scientist would be credited with, say, the discovery of a vaccine.

"That's more of where the future of biomedical research is going what's good for the good of mankind, not me personally," Verdile said.

News reports in recent years have focused on the ethical aspects of CRISPR technology, which in addition to its potential to prevent devastating diseases, could also be used for cosmetic purposes or have unintended consequences that affect the descendants of the person whose genes are edited. The Albany Prize Committee did not consider such "what if" scenarios, Verdile said, leaving those conversations for future ethicists and policymakers as specific medical techniques are developed.

The Albany Prize, one of the nation's largest for science and medicine, was established in 2000 by the late Morris "Marty" Silverman, a New York City businessman and philanthropist who grew up in Troy. A commitment of $50 million from the Marty and Dorothy Silverman Foundation allows for the prize to be awarded annually for 100 years.

Albany Med released the 2017 award recipients' names Tuesday morning. The recipients will formally receive their awards at a Sept. 27 ceremony in Albany.

chughes@timesunion.com 518-454-5417 @hughesclaire

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Gene-editing scientists to share $500K Albany Med prize - Albany Times Union