October 31, 2020

The Niche

Knoepfler lab stem cell blog

gene therapy

5 min read

There are many genomics meetings out there these days, but The Future of Genomic Medicine meeting (#FOGM18) at Scripps in La Jolla is one of my favorites. This meeting is uniquely empowering. The people and the talks combine for a one-of-a-kind experience. The venue doesn’t hurt either at the Scripps Seaside Forum. The evening before the meeting I walked from the hotel to the venue and took a picture from below at sunset. If you look up by the palm trees you can see hang gliders …Read More

5 min read

The news that CRISPR-Cas9 gene editing in its current form may not work in a substantial fraction of people due to many of us having immunity to Cas9 came as a shock to many, but if you think about it, maybe it’s not so surprising. I don’t see it as the end of the world. A (preprint) from a group led by Matthew Porteus started this lively discussion a few days ago. The preprint is entitled, “Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans.” Some people are freaking …Read More

4 min read

Epidermis regeneration is an important approach to helping patients with a variety of serious skin problems that can arise due to genetic conditions or injuries such as severe burns. An exciting new Nature paper reports epidermis regeneration on a strikingly large-scale. The child who is the subject of the study suffers from a sometimes fatal genetic skin disease called junctional epidermolysis bullosa (JEB). In fact, in the particular case described in the new paper, the patient would have likely died without some transformative intervention given …Read More