CRISPR Archives - The Niche

Weekly reads: Chinese CRISPR biotechs, cultured meat war, MSCs for Alzheimer’s

March 23, 2025 / Professor Paul Knoepfler, Ph.D. / 6 Comments

HuidaGene Therapeutics, Chinese CRISPR biotech

Chinese CRISPR research continues to grow and this includes interesting CRISPR biotech firms. Some here in the U.S. worry that American gene editing firms could be lagging behind. Especially over the past year, investors and even some scientists have voiced frustration. U.S. gene editing firms like Editas have been struggling for years. Its stock is […]

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Weekly reads: Verve Therapeutics, Casgevy UK OK, GDNF, MYC

November 19, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Some folks can view data from early, small clinical trials too skeptically or overly enthusiastically, and maybe that’s going on with some preliminary results from Verve Therapeutics. Good news? Bad news? I’m going to start with two articles about the same news that have very different vibes. What do I think? On the whole, I

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Weekly reads: CRISPR sickle cell, Parkinson’s, pig-human chimera concerns

September 10, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

CRISPR gene editing has made rapid progress heading from bench to bedside. Perhaps the fastest has been its progress toward clinical use to combat sickle cell disease. We’ll start with a new paper on one major effort here. CRISPR gene editing. This process often involves cutting DNA, which then can be used as an opening to

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Weekly reads: big CRISPR mtg, Mesoblast good news, Neuralink spiked

March 12, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Where do things stand with potential applications of CRISPR and other gene editing technologies in patients? Overall, things are looking very positive. CRISPR human trials This week the third big international human genome editing meeting took place in London. The summit addressed numerous potential clinical applications. It was good to see the agenda included quite

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Weekly reads: human CRISPR, MRT risks, private IRBs, skincare

March 5, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

It’s funny how sometimes there are many new articles about one general topic like this week with heritable (and somatic) human CRISPR gene editing and related tech. There are clear reasons for optimism in the somatic arena given advancing trials. Germline editing remains highly questionable in my view even just technically. Then there are loads

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Updates on CRISPR therapy development and clinical trials

November 22, 2022 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

When I first heard of CRISPR gene editing I was excited for my lab to try it out, but it seemed a long way from the field having even one CRISPR therapy available to treat human disease. In the Knoepfler lab, we study genetic and genomic programming in human development and disease. Gene editing seemed

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Weekly reads: aging, CRISPR delivery, hair, oocytes, paralysis

January 23, 2022 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

The big news of the week was the launch of Altos labs and their ambitious plan to tackle aging through cell therapies. Altos has recruited a large number of top cell biologists away from academia. That team plus top biotech execs and a $3B war chest make Altos one to watch in coming years. I

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Clade Therapeutics pursues universal cells for cancer therapy

November 4, 2021 / Professor Paul Knoepfler, Ph.D. / 2 Comments

Clade Therapeutics has been pursuing the idea of using gene-editing to make universally compatible cells. The potential here is big. What if you could make cloaked stem cells that could be used to produce cellular therapies that could be given to most patients without fear of immune rejection? Clade Therapeutics Boston Business Journal covers the

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Ukraine clinic seems to backpedal from CRISPR people idea

April 30, 2021 / Professor Paul Knoepfler, Ph.D. / 1 Comment

medeus clinic doctors. their recruitment emails had suggest a goal to crispr people for trait enhancement.

I wrote earlier this week about Medeus clinic in Ukraine seemingly trying to recruit genetics researchers to CRISPR people for trait enhancement. Aiming for CRISPR people with trait enhancement? In the recruitment emails they sent out it seemed fairly clear that they were planning to do CRISPR on people somehow via stem cells. They mentioned

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Ukraine clinic plans to sell CRISPR enhancements: hair color, skin, & breast size

April 28, 2021 / Professor Paul Knoepfler, Ph.D. / 3 Comments

It was a couple of years back that researcher He Jiankui claimed he made three “CRISPR babies.” No one would think about doing any kind of rogue gene-editing again, right? Not even somatic gene edits. After all, as far as we know he’s in jail. We still don’t know the health risks that these three

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