October 31, 2020

The Niche

Knoepfler lab stem cell blog

gene editing

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

5 min read

As a pivotal advance in the gene-editing field and timeline, CRISPR continues to be utilized for research on stem cells and human diseases. Today, I will explain some of the most notable recent findings in the stem cell-CRISPR field. To start things off, I have also created an infographic that briefly explains what CRISPR-Cas9 is, what stem cells are, and how they both can be used together to analyze human disease through genetics and potentially treat some specific diseases in the future. You may …Read More

5 min read

Two prominent scientists, Robin Lovell-Badge and George Daley, have been amongst the most outspoken proponents of leaving the door open to heritable human genetic modification via CRISPR. While they each have articulated their reasons in somewhat different ways at times, their core reasons arguing in favor of future heritable CRISPR appear largely the same. In this post I tackle each of these arguments in favor of leaving the door open to “CRISPR babies” with science-based counterarguments. I also raise larger risks to going down …Read More

3 min read

What happens when one of the most exciting stem cell biotechs ViaCyte teams up with a firm like CRISPR Therapeutics to use a combination of gene editing and cell therapy? Hopefully a synergistic partnership emerges that in this case can develop an effective stem cell-based therapy for diabetes. You can see a press release on the news. (Update, see my new interview after the news broke with ViaCyte CEO Paul Laikind) ViaCyte has been a frequent subject of posts on The Niche related to …Read More