January 15, 2021

The Niche

Trusted stem cell blog & resources

gene editing

4 min read

The paper of the week reports using base-editing, a kind of gene-editing, to reverse mutations associated with rapid aging syndromes, generally called progeria, but there are a lot of other interesting pubs to recommend for reading this week. I go over it all in this post. Gene-editing to fight premature aging syndromes In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice, Nature. This work comes from a team led by David Liu. It rightly made a big splash in the media. While it’s …Read More

3 min read

How should we view biohackers and their DIY use of CRISPR outside of the traditional industry or academic lab environment? Introduction to “Biohackers” and DIY CRISPR Who are biohackers exactly? They are people who “hack” their own biology by transforming their bodies through the insertion of technology or modification of their DNA. More rarely, biohackers hack other people’s bodies, but that could change and become more common. In recent years, with the strides made in CRISPR technology, biohacking in genome modification has garnered much …Read More

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

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

3 min read

“If I’m going to the trouble of cloning myself, I want the clone to be a copy of me!” I’m imagining what someone might say if they were told that their expensive and ethically dubious personal cloning efforts produced a clone that was somebody else instead of them. Even if the clone was very similar to the clonee, perhaps like a sibling who was nearly but not quite an identical twin, the clonee might be totally PO’d. When I think about “human “cloning” I …Read More

5 min read

There are many genomics meetings out there these days, but The Future of Genomic Medicine meeting (#FOGM18) at Scripps in La Jolla is one of my favorites. This meeting is uniquely empowering. The people and the talks combine for a one-of-a-kind experience. The venue doesn’t hurt either at the Scripps Seaside Forum. The evening before the meeting I walked from the hotel to the venue and took a picture from below at sunset. If you look up by the palm trees you can see hang gliders …Read More

5 min read

The news that CRISPR-Cas9 gene editing in its current form may not work in a substantial fraction of people due to many of us having immunity to Cas9 came as a shock to many, but if you think about it, maybe it’s not so surprising. I don’t see it as the end of the world. A (preprint) from a group led by Matthew Porteus started this lively discussion a few days ago. The preprint is entitled, “Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans.” Some people are freaking …Read More