January 27, 2021

The Niche

Trusted stem cell blog & resources

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

9 min read

Human germline CRISPR raises major bioethical considerations, but what about technical issues? Setting aside the many ethical issue about the general idea of human modification itself, could this really work? Yes in theory it could, but there are some very tough technological challenges that could and likely would cause failures or unacceptable outcomes at many steps along the way. These failures or unacceptable outcomes could easily involve real, live people who could be harmed or die. It’s very different than simple in vitro research so …Read More

4 min read

Some months back a USPTO court issued a ruling that most interpreted as meaning the Broad Institute had won the so-called ‘CRISPR patent battle’ in the U.S. and that UC Berkeley, Jennifer Doudna, and Emmanuelle Charpentier had lost. Now this week Berkeley has appealed that ruling. It seems the odds are against Berkeley prevailing in its appeal, but frankly Berkeley deserves the main CRISPR patent and Broad doesn’t. Interestingly, the European Patent Office apparently agrees with this view and disagrees with the USPTO. Update: note that the Berkeley patent …Read More

2 min read

What is the future of Genome Medicine? The meeting by that same name that I’m at down here in La Jolla is all about tackling this question and the line up of speakers today on the first day is amazing. I’m speaking about IPS cells as a basis for personalized medicine tomorrow morning so that’s exciting. As time permits I’m going to try to do a bit of stream of consciousness live blogging of the event so here goes. Don’t expect everything to be a …Read More

1 min read

So everyone is buzzing about the CRISPR patent court decision (which BTW I think was flawed but that’s for another post), but the research roars on at warp speed. Here are 7 recent CRISPR articles that caught my attention. What are your favorite recent CRISPR papers? Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration. Do you think the term “genome surgery” is appropriate? Efficient CRISPR/Cas9-assisted gene targeting enables rapid and precise genetic manipulation of mammalian neural stem cells. CRISPR on the brain. Muscle-specific CRISPR/Cas9 …Read More

3 min read

Last year I heard from several sources that there somewhere between 3-5 unpublished manuscripts reporting the use of CRISPR gene targeting in human embryos being shopped around at various journals in addition to the one that had been published. Since that time we’ve seen a grand total of one additional paper reporting on CRISPR of human embryos. So what gives? Were the sources wrong? I don’t think so and I believe there are additional labs pursuing research on the use of CRISPR in human embryos. …Read More

2 min read

When potentially game changing new technologies are reported such as NgAgo gene editing, both scientists and the public get excited, but especially if such new reports stem from a single paper it is wise to take a cautious approach for a while. The key question is whether the new findings will turn out to be reproducible. With the case of NgAgo specifically, the Nature Biotechnology paper reporting potentially very desirable gene editing properties, drew a lot of interest. See archived blog posts on NgAgo here. …Read More