January 19, 2021

The Niche

Trusted stem cell blog & resources

CRISPR sickle cell

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

3 min read

By Anna Everette The title of this article is partially borrowed from a fascinating Lightning Presentation delivered by John Doench of Broad Institute at CRISPRcon this year. In his talk, Mr. Doench pointed out how we’ve been looking up to this promising technology for a while now, hoping it will deliver the anticipated results (see image below): And for a second consecutive year, people from different walks of life gathered at this event to figure out how they could contribute to the discussion. But …Read More

2 min read

What is the future of Genome Medicine? The meeting by that same name that I’m at down here in La Jolla is all about tackling this question and the line up of speakers today on the first day is amazing. I’m speaking about IPS cells as a basis for personalized medicine tomorrow morning so that’s exciting. As time permits I’m going to try to do a bit of stream of consciousness live blogging of the event so here goes. Don’t expect everything to be a …Read More