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Glenn Nichols, surrounded by his hospice team. The author is in yellow.

Genetic Linkage

A Conversation with CRISPR-Cas9 Inventors Charpentier and Doudna

December 2, 2015

Tags: CRISPR-Cas9, gene editing, genome editing

CRISPR-Cas9 works like scissors on double-strand DNA. (NHGRI)
At the American Society of Human Genetics meeting in October, CRISPR-Cas9 inventors Jennifer Doudna and Emmanuelle Charpentier accepted the Gruber Genetics Prize, then stopped by the press room. For me, this was a little like sitting down with Bono and Bruce Springsteen, but the women were wonderfully down-to-earth, and a little stunned at all the attention since they published their key paper in 2012 on the technique that is speeding gene editing and making genome editing a reality.

This week an International Summit on Human Gene Editing held in Washington DC discussed the potential promises and pitfalls of gene editing technology. A terrific review is here. For those of us who were around at the debut of modern biotechnology in the 1970s, it’s déjà vu all over again. I hope the outcome will be the same. Although concern over recombinant DNA technology back then began with alarm, it basically ended with not triple-headed purple monsters, as my then-grad-school advisor dubbed the concern, but with a new and more targeted source of drugs, beginning with human insulin.

Below are selected comments from Drs. Doudna (a Howard Hughes Medical Institute Investigator and professor of molecular and cell biology and chemistry at the University of California, Berkeley) and Charpentier (director of the new Max Planck Institute of Infection Biology in Berlin) from their talks and visit to the press room in October. I’ll cover here what I didn’t a few weeks ago here and in Medscape to accompany the conference. (more…)

Will Layla Save Gene Editing?

November 14, 2015

Tags: gene editing, CRISPR, TALENs

(Great Ormond Street Hospital)
I had planned to blast last Thursday’s news of the use of gene-editing to save a British baby from aggressive leukemia. “Two months later, Layla was cancer-free,” proclaimed one of many enthusiastic reports.

I’m always skeptical when I hear the words “cancer” and “cure” in the same sentence, let alone uttered so soon after treatment and without an accompanying technical paper so I can see the data. But when I considered the timing of unfolding events, I realized that the seemingly premature reporting of Layla’s rapidly restored health just might add an important point to the heated discussion over gene and genome editing. That is, can we keep the promising clinical applications on somatic cells, while forbidding the Frankenstein scenarios of germline manipulation? (more…)

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A spectacularly-illustrated, clearly written human anatomy and physiology textbook, used in pre-health profession programs throughout the U.S.
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