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Gene editing pioneers win Nobel Prize in chemistry

Jennifer Doudna and Emmanuelle Charpentier won the Nobel Prize in chemistry on Wednesday for their work developing the gene editing tool CRISPR.

Why it matters: Gene editing could transform biology and medicine with its wide-ranging applications for understanding and treating disease, optimizing crops and eradicating pests. But its potential use in treating human diseases by changing genes that can be inherited raises major ethical questions that will challenge scientists for decades.

The backdrop: Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR, are sequences of genetic code that bacteria evolved to find and target invading viruses.

  • In 2012,Doudna, a biochemist at the University of California, Berkeley and Charpentier, a microbiologist now at the Max Plank Institute for Infection Biology, reported CRISPR can be programmed to lead enzymes to genetic sequences that the enzyme then precisely snips or edits, turning it on or off or changing its function.

Between the lines: Doudna and Charpentier have been considered top candidates for the prize for several years but there's an ongoing fight over patents for CRISPR and its use.

  • Other researchers, including Feng Zheng of the Broad Institute at MIT, have made key contributions to gene editing as well but were not recognized by the Nobel committee.
  • Berkeley and MIT have battled for years over the patent rights to CRISPR, though it's unclear how the Nobel will affect the legal fight.

What to watch: Earlier this week, Doudna launched Scribe Therapeutics, a startup that aims to use engineered CRISPR molecules and delivery technologies to edit cells while they are in the body. (Other approaches remove cells from the body, edit them and reintroduce them.)

  • Scribe, which is collaborating with Biogen, is looking to treat amyotrophic lateral sclerosis (ALS) first, per FierceBiotech.

Of note: "The award smashed records and made scientific history as the only science Nobel ever won by two women," Sharon Begley writes in STAT.

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