January 17, 2021

The Niche

Trusted stem cell blog & resources

weekly reads

4 min read

I’m playing catch-up on some reading given how busy I’ve been and this includes a groundbreaking NEJM pub on CRISPR for Sickle Cell and Thalassemia. CRISPR for Sickle Cell From December, here’s the key paper in the NEJM: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. There’s a lot to like about this clinical trial paper and it gives real hope to patients with Sickle-Cell Disease and Thalassemia. It takes an interesting, indirect clinic approach to addressing Sickle Cell by turning off a …Read More

4 min read

The paper of the week reports using base-editing, a kind of gene-editing, to reverse mutations associated with rapid aging syndromes, generally called progeria, but there are a lot of other interesting pubs to recommend for reading this week. I go over it all in this post. Gene-editing to fight premature aging syndromes In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice, Nature. This work comes from a team led by David Liu. It rightly made a big splash in the media. While it’s …Read More