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”