gene editing Archives

Dissecting that Neanderthal brain organoids Science pub

February 15, 2021 / Professor Paul Knoepfler, Ph.D. / 4 Comments

Organoids and especially brain organoids, which are made from pluripotent stem cells, are one of the most interesting developmental biology technologies of the last half a dozen or so years. Still some folks can’t help but get carried away when thinking about brain organoids it seems. A new Science paper from a team led by

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Weekly reads & paper of the week: gene-editing vs. aging

January 10, 2021 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

koblan et al 2020 nature gene editing progeria

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

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Can Gene-Edited Stem Cells Treat Cystic Fibrosis?

September 28, 2020 / Ricki Lewis / Leave a Comment

human-IPS-cells-made-in-Knoeplfer-lab-stained-for-TRA-160

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

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Counterpoints to Lovell-Badge & Daley’s CRISPR baby rationales

February 11, 2019 / Professor Paul Knoepfler, Ph.D. / 5 Comments

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

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ViaCyte & CRISPR Therapeutics team up to battle diabetes

September 17, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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

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Surprising reason why human cloning may produce someone else

July 5, 2018 / Professor Paul Knoepfler, Ph.D. / 4 Comments

“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

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Highlights FOGM18 Day 1: Data from Ancient Dirt, Genomics Dilemmas, CAR-T, CRISPR

March 2, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

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

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CRISPRcon guest post: Hope for a brighter future

August 19, 2017 / Professor Paul Knoepfler, Ph.D.

By Anna Everette “In our society, we have an addiction to vertical genetic transmission. It’s called sex and having a child who looks like you”. This was perhaps the most memorable quote from the event’s keynote speaker, Greg Simon, Director of the Biden Cancer Initiative. It also happens to highlight the most compelling reason to

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Poll: Will NgAgo strongly challenge CRISPR in coming years?

July 25, 2016 / Professor Paul Knoepfler, Ph.D.

Will NgAgo strongly challenge CRISPR in coming years? Take our poll and also see the results to find out what others thought. 2020 update: NgoAgo has ending up going nowhere, but 4 years ago we didn’t know that would be it’s fate although some of us were skeptical early on.

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gene editing

Recommended reads: click editing, CRISPR v. progeria, diapause transcriptional program