CRISPR’s next target is the fetus genome

The goal is to treat unborn children as early as possible, before their disease causes irreversible damage. But the ambition is to do so without heritable DNA changes, that is, by targeting only somatic tissues and avoiding sex cells. Fetal genome editing, then, differs from embryo editing, which has raised so much controversy in recent years. The best way to understand how far it has come and how much remains to be done is to tell the story of the scientist most committed to this challenge. The opportunity is provided by a longread published in STAT, where Tippi MacKenzie’s biography is interwoven with a review of the field.

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Happy 10th Birthday CRISPR!

The seminal paper by Doudna & Charpentier was published online at the end of June 2012. The printed issue came out a few weeks later, on August 17 (don’t try to buy it, Science VOLUME 337|ISSUE 6096 is out of stock). No wonder the gene-editing community is in the mood for celebration these days. If you are too, don’t miss the chance to read these articles on CRISPR’s ten-year anniversary!

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CRISPR-based Covid tests: what’s going on?

Doudna’s creature (Mammoth Biosciences) and Zhang’s company (Sherlock Biosciences) are developing CRISPR-based coronavirus tests similar to a home pregnancy test: portable, cheap, fast, and simple. Both will be easily adapted to detect any new emerging virus. Both received emergency use authorizations in the US in the fall of 2020 and hope to enter the market by the end of 2021. According to this piece by Walter Isaacson, competition is hot but all the intellectual property questions have been put aside for common good.

CRISPR rising stars

Andrew Anzalone (Broad Institute), Jennifer Hamilton (Berkeley) and Cameron Myhrvold (Princeton)

December is time for rankings and forecasts. Let’s start with STAT News celebrating young talents who could become the next generation of scientific superstars. Three CRISPR researchers appear among STAT wunderkinds. As a postdoc at the Broad Institute, Andrew Anzalone helped make a key advance by developing prime editing, where the same RNA molecule specifies the target and the desired edit. Jennifer Hamilton, from Berkeley, works on solving one of the major hurdles of CRISPR-based therapies: delivering the genome editor to the desired cells. Cameron Myhrvold, has since worked at the Broad Institute on developing CRISPR-based diagnostics such as CARMEN and is about to start his own lab at Princeton.

Knocking out cholesterol

Consider this scenario, depicted in Nature a few years ago. “It’s 2037, and a middle-aged person can walk into a health centre to get a vaccination against cardiovascular disease. The injection targets cells in the liver, tweaking a gene that is involved in regulating cholesterol in the blood. The simple procedure trims cholesterol levels and dramatically reduces the person’s risk of a heart attack”.

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Picks of the week

The ethics of using CRISPR to improve the odds of savior siblings. This is when a couple tries to have another baby who is both healthy and a suitable donor “match” for their older kid (“Could editing the DNA of embryos with CRISPR help save people who are already alive?“, STAT News, Sept. 16).

Carl Zimmer explaining a CRISPR experiment carried out to understand why many cancer drugs fail (“Why Aren’t Cancer Drugs Better? The Targets Might Be Wrong”, New York Times, Sept. 11; see also the paper in Science Translational Medicine)