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Small positive step for BlueRock Therapeutics Stem Cell Therapy for Parkinson’s

August 28, 2023 / Professor Paul Knoepfler, Ph.D. / 4 Comments

BlueRock Therapeutics, Parkinson's disease, brain imaging

Several groups including Viviane Tabar who is a founding scientist at BlueRock Therapeutics are moving forward on potential cell therapies for Parkinson’s Disease. BlueRock just released some new data that’s worth a look. Check out the new BlueRock poster. In today’s post, I briefly discuss the new data. I also go into the larger context […]

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Weekly reads: reprogramming hearing loss, heart disease, eye drops, sickle cell

April 30, 2023 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

It’s mostly been a week of good and encouraging news in the regenerative medicine space including with gene therapies maybe with the exception of some eye drops warnings (more below). There’s realistic hope for an approved sickle cell disease soon. I also see some long-term positive news on hearing loss research. Hematopoietic stem cell transplant

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Weekly reads: UC Davis Medical School diversity, CRISPR, Parkinson’s

March 26, 2023 / Professor Paul Knoepfler, Ph.D. / 2 Comments

It’s been almost seventeen years that I’ve been a professor here at UC Davis Medical School. It feels like home. I enjoy teaching our first-year medical students each year. Some end up doing research in my lab. Our school recently got a nice write-up over at STAT News by Usha Lee McFarling on the diversity

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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: stem cell good news, bad news on sickle cell, MS, IPSCs

January 8, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

victoria gray family crispr sickle cell b

Let’s start with some stem cell good news on evolving approaches to treat sickle cell disease involving stem cells. Then on another related front things were more concerning. Stem cell good news on sickle cell disease, a complication Sickle Cell Disease News reports Adding Briquilimab Boosts Success of Sickle Cell Stem Cell Transplant. This is a

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2023 stem cell & regenerative medicine predictions

December 28, 2022 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Toward the end of each year, I make stem cell & regenerative medicine predictions for the coming year. It’s a fun tradition here on The Niche that goes way back. The stem cell field has changed dramatically as reflected in each year’s predictions. Here’s a list of past predictions with my grades of how they

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He Jiankui says he’s back in the lab after prison for CRISPR babies

November 29, 2022 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

Earlier this year Chinese researcher He Jiankui finished his prison sentence. When I saw that news I wondered about the next chapter for him. It seems he’s already back in the lab doing genetics research. At least that’s according to a blog by He Jiankui himself. Can we trust that what he has written on his website is

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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 stem cell reads: space, plagiarism, HIV, diabetes, more

July 31, 2022 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

I took a cross-country road trip earlier this month that was pretty amazing and barely thought about stem cell research for the first time in years. We did run across one stem cell clinic in Florida by accident. I’ll write about that trip soon, but first we have our weekly reads including some very cool

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Grading my 21 stem cell & regenerative medicine predictions for 2021

December 22, 2021 / Professor Paul Knoepfler, Ph.D. / 3 Comments

The world of stem cell research and regenerative medicine is unpredictable but it’s fun to be a part of it and try to do predictions. Each year in late December or early January I make prognostications for the coming year for stem cell research and regenerative medicine. I made 21 such predictions for the current

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