Back to the basics of xenohearts

UHeart™ (photo credit United Therapeutics)

As you probably know, on January 7 at the University of Maryland Medical Center in Baltimore  a 57 years old man named David Bennett became the first human to have his heart replaced with that of a CRISPRed pig. But what does make a xenoheart suitable for transplantation?

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MetaCRISPR, how to edit microbiomes

Jill Banfield and Jennifer Doudna (photo by Keegan Houser)

The best way to summarize the new metaCRISPR approach, recently published in Nature Microbiology, is the Twitter thread by Jill Banfield:

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CRISPR children, how are they?

Credit: Stefano Navarrini, Innovative Genomics Institute, Anna Meldolesi

Lulu and Nana are three years old. Amy is the name Nature Biotechnology uses to refer to the third CRISPR baby, born in late spring-early summer 2019. Their health is a closely held secret, that Vivien Marx has investigated for the journal’s December issue. “A full understanding of the health risks faced by the children due to their edited genomes may lie beyond the reach of current technology”, she writes. Despite or maybe because of that, the news feature is well worth reading. Below are a few points:

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Base-editing comes of age and more SCD news

Alexis Komor and Nicole Gaudelli developed based editing when they were postdoc in David Liu’s laboratory at Harvard. Credit: The CRISPR Journal

The first Investigational New Drug (IND) application for base-editing technology has been cleared by the Food and Drug Administration. BEAM-101, developed by Beam Therapeutics, is an ex vivo base-editing product candidate, meaning that it uses a modified form of CRISPR capable of making single base changes without double-stranded DNA cleavage.

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Would you buy these apples?

Three scab-resistant apples resulting from conventional breeding at the University of Bologna in Italy. I got them yesterday from prof. Stefano Tartarini at a workshop on NBTs (New Breeding Techniques, also called TEA in Italy, meaning Assisted Evolution Techniques). I filled out the evaluation questionnaire on the red, the pink, and the rusty apple. The last question is: would you buy it if it was a TEA fruit? A big yes from me! (My favorite is the rusty one).

Drill & thrill, first CRISPR wheat sown in UK

Rothamsted scientist Nigel Halford tweeted this photo on October 26 with the comment: “Great to watch our field trial of CRISPR low asparagine/low acrylamide wheat being drilled today.”

The picture shows the first field trial of CRISPR wheat in Europe. Starting a couple of weeks ago, the trial will run for five years, with plants being sown each October and harvested the following September.

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Fyodor Urnov imagines CRISPR cures

Fyodor Urnov, University of California, Berkeley/Innovative Genomics Institute. Urnov is a pioneer in the field of genome editing, with a diverse background in academia, industry and the nonprofit sector. During his time at Sangamo Biosciences he co-developed and co-named human genome editing.

“Imagine CRISPR cures” is the title of the keynote by Fyodor Urnov at the World CRISPR Day conference on October 20. The talk was far from a celebration: “The fact that editing represents an approach to the majority of monogenic diseases in principle doesn’t mean that some biotech will take on disease number 823 in practice, and there are over 5,000 monogenic conditions on OMIM. Three years to IND in the best case scenario and cost scale of more than $6m per disease, that’s incompatible with either the promise of CRISPR to edit any given mutation which it can do or the unmet medical needs”. Don’t miss the on-demand video to learn more about the challenge of N=1 trials and Urnov’s call to arm against ultra-rare diseases.

Xenotransplantation: time to go deeper

Photo credit: Joe Carrotta

And so it happened. “In a first, surgeons attached a pig kidney to a human, and it worked,” as the New York Times puts it. Data are scarce, however, and all the info we have is from the general media. The kidney came from a GalSafe pig, which is the only one FDA approved so far. But scientists from several companies have already developed pigs much more engineered than that (with three or four porcine genes knocked-out instead of one, and human gene additions). To get an updated picture, we highly recommend this article published in Nature Biotechnology last April.