July 4, 2020

The Niche

Knoepfler lab stem cell blog

gene therapy

2 min read

It’s been a cool week for stem cell and other associated kinds of research. In this post I have a series of links to a variety of interesting developments and papers. Gene therapy cures bubble boy infants. William Wan over at the WaPo on this good news. Nope, this wasn’t done with CRISPR, but rather with a viral transgene approach (not TALENs as I mistakenly had originally written here). “The pigs were dead. But four hours later, scientists restored cellular functions in their brains”. This …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

8 min read

What’s Cambrian Genomics? With rapid advances in genomics, gene editing, and methods to reproduce life in the lab such as cloning, today creating genetically modified organisms (GMOs) has never been easier. While there have been of course loads of discussions of GMOs in the past, we are entering a new era of what one might call DIY GMOs, when everyday people outside of a lab (possibly enabled by biotech companies) might be able in theory to create new GMOs themselves. This could in the …Read More