August 15, 2020

The Niche

Knoepfler lab stem cell blog

Paul Knoepfler

5 min read

The biotech Mesoblast is starting an expanded access study of its stem cell product for kids with a severe offshoot of COVID-19. The cellular drug in this case is remestemcel-L (more below). While most children fare better than adults in dealing with COVID-19, the rare subset of kids with COVID-19 for this study have more severe disease than the average COVID patient and some die. Physicians need more potential treatment options here for these children. In that sense one could argue that testing more new …Read More

3 min read

My lab’s new paper in Communications Biology focused on high-grade pediatric glioma that have mutant histone variant H3.3 and we did something fairly novel that we are calling reciprocal CRISPR. Kids with these tumors have a near zero survival rate within a few years of diagnosis so we as a field desperately need something new to give them and their families real hope. Our paper is entitled, “Reciprocal H3.3 gene editing identifies K27M and G34R mechanisms in pediatric glioma including NOTCH signaling.” What we did in …Read More

4 min read

What’s new in the stem cell, cell therapy, and regenerative medicine world as well as biomedical science more generally including cancer? There’s quite a bit of news as reflected in media pieces and new pubs. Today’s post is focused on pubs that just came out. For last week’s recommended reads see here. Oct4-Sox2 Nucleosome Binding & Impact in mESCs This is one of my favorite papers of 2020 so far, coming to us from Science. Mechanisms of OCT4-SOX2 motif readout on nucleosomes. I knew from …Read More

3 min read

Clinical trials using organoids are still rare and a Clinicaltrials.gov search for such organoid work largely brings up in vitro studies. A new clinical study in Japan where organoid material will actually be transplanted into patients is causing a lot of excitement. Masayo Takahashi (高橋 政代) is a pioneer in stem cell-based clinical trial work and more specifically induced pluripotent stem cell (IPSC)-based trials. Her new clinical study is another groundbreaking step in that it is organoid based, in this case for Retinitis pigmentosa. …Read More