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 Medicineon 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).
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.
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.
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.
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 →
It is Science but it could be mistaken for The CRISPR Journal. The latest issue indeed runs three papers by three CRISPR aces – David Liu, Jennifer Doudna, and Feng Zhang – about the cutting-edge fields of biological recorders and advanced diagnostic tests. 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.