July 9, 2020

The Niche

Knoepfler lab stem cell blog

CRISPR-Cas9

4 min read

If it seems to you like dramatic cautionary tales about CRISPR accompanied by all sorts of media are coming at us more frequently, it’s not your imagination. In the latest yesterday, it was reported in a new paper led by Allan Bradley that CRISPR-Cas9 results in sometimes large-scale chromosomal lesions at or even away from the target locus. The media have really sunk their teeth into this one, just as in past situations where CRISPR has reportedly encountered anything from bumps in the road to …Read More

1 min read

Heritable CRISPR to be tried in humans sooner or later…or never? Will someone somewhere in the world try to use CRISPR gene editing or related technology to introduce heritable genetic changes into actual human beings in the next decade? I’m not talking about gene editing viable human embryos just for research which is already ongoing, but rather CRISPR’ing human embryos to then use to try to make babies who grow up to be adult humans with a specific genetic change. How likely is this? Take …Read More

4 min read

Some in CRISPR-Cas9-land who are focused on potential future clinical applications are kind of rejoicing or at least sighing a breath of relief. This upbeat swing in the atmosphere (from investors especially) was sparked by retraction of that paper, the one initially reporting tons of supposed off-target CRISPR-Cas9 activity in mice, which turned out to be a “nothing burger” according to one investment site. Off-target activity definitely still needs to be on people’s radar screens, but it’s a problem that’s not nearly so widespread as that paper incorrectly …Read More

3 min read

There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat expansions in the regulatory region of the FMR1 gene and associated epigenetic alterations including DNA methylation that tend to shut off gene expression. The new paper, Liu, et al., …Read More