September 28, 2020

The Niche

Knoepfler lab stem cell blog

CRISPR

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

3 min read

Something called, “The International Commission on the Clinical Use of Human Germline Genome Editing” has issued a new report on heritable human genome editing (HHGE). The Commission was convened by the U.S. National Academy of Medicine, the U.S. National Academy of Sciences, and the U.K.’s Royal Society. This post is my initial take on the summary of the lengthy report. I hope to have time to read the full report in coming weeks and it’s possible some new perspectives may emerge. Here is my thinking as …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