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).
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.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
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.
Click the links below to discover Ddda, the exceptional enzyme that allows mitochondrial editing, and celebrate curiosity-driven research.
The Nature paper by Joseph Mougous and David Liu: “A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing”
The news: “Scientists make precise gene edits to mitochondrial DNA for first time”
The news and views: “Mitochondrial genome editing gets precise”
The editorial: “Mitochondrial genome editing: another win for curiosity-driven research”
European scientists must wait for the EC to carry out targeted consultations with Member States and EU-level stakeholders. Then the Commission study on new genomic techniques will be delivered by 30 April 2021. In the meantime, the United States has decided to relax the rules for biotech crops.Continue reading
Do you remember prime editing? It’s the new ‘search-and-replace’ genome editing technology that mediates targeted insertions, deletions, all 12 possible base-to-base conversions, and combinations thereof. The first good news is that David Liu et al. adapted prime editors for use in rice and wheat, so don’t miss their paper in Nature Biotechnology.Continue reading
Great piece of science by the Liu Lab in Nature, describing the brand new “Search-and-replace genome editing without double-strand breaks or donor DNA”. How is the CRISPR community reacting?
Best quote: “One of those ‘Yay, science!!!’ kind of moments” (Fyodor Urnov quoted in Science)
Most ironic: “Congratulations @davidrliu. We’d probably have published this paper as well (The CRISPR Journal tweet)
Best title: Genome Editing Heads to Primetime (Genetic Engineering and Biotechnology News)
Most understated: A New Gene Editing Tool Could Make CRISPR More Precise (Smithsonian Mag)
Most hyped: A New CRISPR Technique Could Fix Almost All Genetic Diseases (Wired)
CRISPR needs to anchor itself near a short sequence called PAM to do its job. In the book “Modern Prometheus” (Cambridge University Press) James Kozubek says a PAM is like a shoehorn, where the Cas9 nuclease begins to clasp down to recognize the right site and cut. In order to fit every gene, a super-adjustable shoehorn would be needed. Think of it as the equivalent of a bump key that can open any door. A Broad Institute group led by David Liu has almost reached the goal with xCas9, the new super-adjustable Cas9 variant described in Nature this week. Continue reading