Natural evolution has had four billion years to experiment with living matter. From now on, artificial intelligence will also help expand the catalog of possible and desirable molecules. These so-called ‘language systems’ are no longer limited to producing text or images, as exemplified by ChatGPT or Midjourney. They can now be instructed and utilized to design new proteins, thereby potentially creating improved CRISPR scissors.
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Anyone interested in advanced therapies is familiar with the acronym CAR-T. These are T lymphocytes modified (also with the help of CRISPR) to better recognize and attack cancer cells, and they have already proven to be a successful strategy for blood tumors. Now hopes are high that a similar approach may also prove useful for multiple sclerosis, which is an autoimmune disease. The idea is to use CAR-Ts to prevent B lymphocytes from attacking nerve cells, including in the brain. The first clinical trial is recruiting patients in the U.S. Read more in Nature.
Let’s come to the use of New Genomic Techniques in crops. The European Commission’s regulatory proposal (approved by the EU Parliament on Feb. 7) excludes the use of edited plants in organic farming, but among organic producers not everyone is against NGTs and this may bode well for a possible peaceful coexistence between the different types of production in the years to come.
Finally, we point out the latest advance in animal editing: porcine virus-resistant pigs. The paper came out in the CRISPR Journal, but you can also read about it in GEN.
Recently David Liu announced that Prime Medicine will likely submit the first human trial application in 2024. The standard version of CRISPR uses an RNA guide to find the editing site in the genome. Prime editing, on the other hand, also uses the same RNA molecule to direct the correction, in short, to specify what to do as well as where to go.
This insight blossomed in Andrew Anzalone’s mind a few years ago during his PhD at Columbia University. The first practical demonstration came with a paper published in Nature in 2019 after joining the Liu’s Lab at the Broad Institute. Since then, this platform has been used in hundreds of experiments to fix all kinds of mutations in vitro and in animal models.
Meanwhile, the company co-founded by Anzalone and Liu has begun work on 18 treatments, the most advanced for chronic granulomatous disease. To learn more, from the eureka moment to the latest developments, we suggest listening to the Close to the Edge podcast and reading Alex Philippidis’ article in GEN.
The news of the week is definitely this: the first clinical trial with base editing (the CRISPR platform used to chemically change single DNA letters without double-strand breaks) hit the goal of lowering cholesterol in patients but raised questions about the risks (with two serious adverse events, including one death), as Nature reports.
But we also recommend reading two other articles. Nature Biotechnology takes a look at experiments using CRISPR to eliminate viruses that manage to hide from the immune system, such as HIV and hepatitis. While Genetic Literacy Project publishes an analysis of the problems that could cripple the new regulation on edited plants proposed by the European Commission and delay (even until 2030) the arrival of the first products on the EU market.
Don’t miss the lipid nanoparticles delivering CRISPR to the lungs (in Nature), the ever-growing CRISPR toolbox (in the CRISPR Journal), and the pig-monkey xenograft that brings experimentation on human patients closer (also in Nature).
Drew Weissman will forgive us, but this will go down in history mostly as Katalin Karikó’s Nobel Prize. And perhaps in addition to being an award to celebrate, it is also an award that should make us angry. Because this scientist’s story is too extraordinary, for all the obstacles she had to face and overcome. It is always said that girls need model female scientists to inspire them, Karikó is a beautiful role model but we sincerely hope that she does not have to be an example to anyone, because it is not fair that a researcher of this stature was forced into precariousness for decades and could not count on a stable professorship in the United States where she moved from Hungary in the 1980s (at the University of Pennsylvania she results “adjunct professor”).
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The European Commission’s proposal for an updated regulatory framework for New Genomic Techniques is due on 5 July, but someone leaked the confidential document online. In a nutshell, if the modification could also have been achieved naturally or by conventional methods, and the plant has the same risk profile as its conventional counterpart, it should be treated similarly to conventional plants and differently from GMOs (it would not require authorisation, risk assessment, traceability, labelling as GMO, but would be placed in a transparency register). For plants in which editing or cisgenesis has led to results that differ from conventional ones, the current GMO rules apply. You can read the Genetic Literacy Project’s explanation here.
Please note a couple of things environamental NGOs and organic producers should like: herbicide-tolerant NGT plants would remain subject to GMO rules, and all NGT plants would remain subject to the prohibition of use of GMOs in organic production.
By now it seems official. The first CRISPR plant to debut in the US market will not be a commodity for industry or intensive livestock farming, as was the case with classic GMOs in the 1990s. This time genetic innovation enters on tiptoe, with a food product designed for discerning consumers. A new type of salad, as nutrient-rich as a wild misticanza but without the bitter notes that usually relegate brassicas to foods to be eaten cooked (see here).
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CRISPeR FRENZY 