A multi-disciplinary panel of 18 experts from all over the world, a two years long consultation, over 150 pages. The much-awaited report of the World Health Organization on human genome editing was delivered on July 12 and is divided into three parts: A framework for governance, Recommendations, and Position Paper. While not legally binding, it is expected to influence both governments and the scientific community, by offering a roadmap based on widely shared ethical principles and usable policy tools.Continue reading
Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicine on 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.
Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).Continue reading
Anti-CRISPR proteins are the rock needed to stop CRISPR-based mosquito-eradicating gene drives, if necessary, and make them safer. In a news feature published last year in Nature, the molecular parasitologist Andrea Crisanti disclosed unpublished data about halting an anti-malaria gene-drive system by adding anti-drive mosquitoes to the mix. “They can completely, 100% block the drive. We can stop the [Anopheles gambiae] population from crashing,” he said. According to the scientist from the Imperial College London, it’s kind of like buying an insurance. Looking ahead to field-testing his sterilization strategy, Crisanti imagined having cages of anti-drive mosquitoes at the ready, just in case things go awry. Well, that work is now published, and anti-drive mosquitoes are a reality. To learn more, see the paper published on June 25 in Nature Communications by Chrysanthi Taxiarchi et al.
The Somatic Cell Genome Editing (SCGE) Consortium is working to accelerate the development of better methods of editing. Seventy-two principal investigators from 38 institutions are pursuing 45 distinct but well-integrated projects, funded by the US National Institutes of Health with US$190 million over 6 years. A perspective published in Nature details their plans:
“New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled—along with validated datasets—into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit—and the knowledge generated by its applications—as a means to accelerate the clinical development of new therapies for a wide range of conditions”.
CRISPR-based diagnostic tests for Sars-Cov2 are coming, as you probably know. But what about CRISPR-based antiviral therapy? It would seem a natural outcome for a technology inspired by the way many bacteria fight their viruses. Indeed this kind of research is being pursued in a handful of labs, using a CRISPR enzyme targeting RNA instead of DNA.Continue reading
CRISPR-based techniques allow the reconstruction of the “family tree” of the cells that compose an animal’s body by marking them with a pattern of deletions and insertions. This kind of barcoding has already helped trace embryo growth and organoid development and is shedding light on essential oncology questions by catching cancer in the act. Read how “Single-cell lineages reveal the rates, routes, and drivers of metastasis in cancer xenografts” in this Science paper and the news from Whitehead Institute.
A paper published in Nature by CRISPR innovator David Liu and a giant in medical genetics, Francis Collins, raises great hopes for treating a rare, devastating pediatric disease causing premature-aging (Hutchinson-Gilford progeria syndrome). “The outcome is incredible,” according to gene-therapy researcher Guangping Gao. “Dance on the lab bench” amazing, according to editing pioneer Fyodor Urnov. Let’s be clear: the CRISPR variant called a base-editor has helped only progeria mice so far, but results are beyond anyone’s wildest expectations. One injection is enough to fix the single-letter mutation in several tissues, doubling mice’s lifespan. To learn more, see David Liu’s tweets and the NIH Director’s Blog.
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.
The Nobel Prize for CRISPR is one of the most exciting ever assigned in chemistry and one of the most celebrated in the media, for reasons related to the invention and the inventors alike. On the one hand, the technique is changing the practice and the image of genetic engineering. On the other hand, Jennifer Doudna and Emmanuelle Charpentier are not merely great scientists; they are a success story in cracking the glass ceiling and a symbol of the strength of collaboration.Continue reading
Covid19 is affecting everyone, but it has hit the sickle cell (SCD) community particularly hard. According to STAT News the pandemic has temporarily stopped clinical trials and the introduction of new drugs, besides directly impacting SCD patients who are at high risk for severe complications from Sars-Cov2 infection and may need hospital assistance for SCD pain crises.Continue reading