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
The rising star of base editing shadowed classic genome editing last week. I’m sure you heard about the ground-breaking papers respectively published by David Liu and Feng Zhang in Nature and Science. CRISPR enthusiasts have probably already enjoyed the piece by Jon Cohen on the new approach, i.e., the rearrangement of atoms in individual DNA letters to switch their identity without even cutting the DNA strands. But let’s take a look also at The Scientist, which runs two must-read articles about the details of the experiments. The first take-home message is the latest achievements are exciting, but base editors are not better than CRISPR, they’re just different. The second one, there is still room for improvement with base editing, and the best is yet to come.