Cholesterol down – ready set edit!

Last week Verve Therapeutics dosed the first patient with a candidate treatment for hypercholesterolemia. This is exciting news for a couple of reasons. First, the technology used: CRISPR 2.0, i.e., base-editing is hitting the clinic (see the news in Nature Reviews Drug Discovery). Second, this is a leap forward into common diseases (“CRISPR for the masses”, says The Washington Post) and a training session for the real challenge, which is to “stop the biggest killer on Earth”, cardiovascular disease (MIT Technology Review).

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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The greatest functional map of all

Credit: Jen Cook-Chrysos/Whitehead Institute

Jonathan Weissman and colleagues used a CRISPR-based method to link each expressed human gene to its function in the cell. Here’s our suggested readings to learn more:
the paper in Cell by Josepg Replogle et al.
the Twitter thread by Joseph Replogle
MIT News (by Eva Frederick) and GenEngNews.

A pig virus in the xenoheart. How bad is the news?

As you probably know, the first patient with a pig heart died two months after the transplant surgery. You probably heard also that a porcine cytomegalovirus (PCMV) may have contributed to the death. However, if you are still wondering how bad is the news for the future of xentransplantation, Linda Scobie from Glasgow Caledonian University is the one to listen to. She leads a research group interested in viral zoonoses in the context of novel technologies such as xenotransplantation, and the contribution and/or reactivation of viruses in chronic disease conditions. Professor Scobie is a member of the World Health Organisation committee for the global consultation on regulatory requirements for xenotransplantation trials. I reached her by email for a feature just published in Le Scienze, the Italian edition of Scientific American. Below you can read her answers.

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Crispy starters from a new journal

Have you heard of GEN Biotechnology? Issue 3 is already out, but I’ve just got my free copy of the inaugural issue (thanks!). Same publisher (Mary Ann Liebert), same executive editor (Kevin Davies), and the same passion for biotech frontiers as The CRISPR Journal. See below some crispy starters from issue number 1:

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Hit&Run – the Italian way to epi-editing

The San Raffaele-Telethon Institute in Milan has been a leading player in gene therapy for many years. Nowadays, Angelo Lombardo, Luigi Naldini, and colleagues are making news with epigenetic editing. Their 2016 paper in Cell on hit-and-run epigenetic editing is considered seminal work. The company they co-founded, Chroma Medicine, received substantial financing, as recently reported by Nature Biotechnology. Last but not least, the presentation given in May at the annual meeting of the American Society of Gene and Cell Therapy inspired a Science news entitled “Better than CRISPR? Another way to fix gene problems may be safer and more versatile.” In brief, they injected mice to silence the expression of the PCSK9 gene, lowering “bad” cholesterol levels for months

The race of Liu’s CRISPR machines

From the base-editing idea first sketched out via email in 2013, to the invention of prime-editing in 2019. From the progeria mutation fixed in mice in 2021 to the upcoming clinical trial for coronary heart disease. The updated story of the most advanced CRISPR tools told by Harvard’s David Liu is not to be missed (here’s the link to the Life Itself conference organized by CNN).

Four tips on epigenetic editing

Credit IGI

The epigenetic way to editing is hot these days. Here are our suggested readings to keep pace:
1) the basics of the tools CRISPRoff and CRISPRon are explained on the website of the Innovative Genomics Institute
2) Nature Biotechnology news on Chroma Medicine, a company pioneering epigenetic editors
3) The Scientist on resetting the DNA of rats to reverse alcohol damage (see also the paper by Bohnsack et al. in Science Advances)
4) the review discussing translational issues in epigenetic editing published by Huerne et al. in The CRISPR Journal.

Picture of the week: Francis Collins’s bars

Francis Collins at the ASGCT 25th Annual Meeting

“Your mission is to make the red bar match the yellow bar”, urges a slide shown by Francis Collins at the annual meeting of the American Society of Gene & Cell Therapy held in Washington. There are almost 7,000 genetic diseases, but only about 500 with therapy. Most are not viable targets in a for-profit setting and won’t be managed by current gene-editing procedures. Hence the call to find something that is scalable. “We need a transformative approach.” Please read Kevin Davies’s account of the inspirational lecture given by the geneticist that led the Human Genome Project, then was appointed director of the NIH, and currently is Joe Biden’s scientific advisor.