November 28, 2020

The Niche

Knoepfler lab stem cell blog

CFTR mutation

6 min read

Drugs that restore the shape of the errant protein behind cystic fibrosis (CF) have, over the past eight years, helped the majority of patients, who have certain mutations. Gene-corrected stem cells might offer a “mutation agnostic” option to CF. CF results from a glitch in a glycoprotein with the unwieldy name “cystic fibrosis transmembrane conductance regulator”, or CFTR. The proteins normally fold into channels that regulate the flow of ions into and out of cells, controlling the balance of water and salts in linings …Read More

2 min read

We’ve all heard the human embryo editing rumors. There is a sense that human embryos have already been CRISPR’d. Rumors are rumors and they may mean not a whole lot, but there definitely is a huge buzz on this right now both in a positive sense and with concern. The excitement is over the power of this technology to do transformative things. The worry is that these papers might easily cross a line or if they are not done incredibly rigorously with unquestionable transparency, institutional …Read More