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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: H3.3 on the brain, Texas bill, HSCs

January 22, 2023 / Professor Paul Knoepfler, Ph.D. / 1 Comment

It’s always exciting when your lab has a new paper and my team just published a study knocking out the H3f3a gene in mice, which codes for histone H3.3 protein. We found that loss of this gene leads to lethality at a late stage of embryonic development. There were also indications of more specific phenotypes

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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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Mesoblast MSCs Quell Peds GvHD; On Road to FDA Approval?

August 20, 2020 / Ricki Lewis / 6 Comments

MSCs-mesenchymal-stem-cells-stromal-cells-small

Graft-versus-host disease (GVHD) is counterintuitive In the weeks and months following a transplant, a major concern is the recipient’s immune system rejecting the “foreign” biological material. But in GVHD, the opposite happens: transplanted tissue unleashes a horde of T cells that spark a cascade of inflammation, within 100 days. Typically, GVHD follows a bone marrow

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Heads up on Hui Yang, another potential aspiring CRISPR baby researcher

January 22, 2020 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Making a CRISPR baby is a controversial idea to even propose now for many reasons, yet even after He Jiankui’s train wreck some people have seemed eager to try it including apparently a scientist whose name perhaps many readers here are not so familiar with in this context: Professor Hui Yang. ‘CRISPR baby guys’ Is

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The Hope of CRISPRcon: Year 2, Guest Post by Anna Everette

June 12, 2018 / Professor Paul Knoepfler, Ph.D. / 1 Comment

By Anna Everette The title of this article is partially borrowed from a fascinating Lightning Presentation delivered by John Doench of Broad Institute at CRISPRcon this year. In his talk, Mr. Doench pointed out how we’ve been looking up to this promising technology for a while now, hoping it will deliver the anticipated results (see

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Updated FDA granted RMAT List (85)

January 10, 2018 / Professor Paul Knoepfler, Ph.D.

Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. Sometimes people call these “r mat” with a space. As of December 12, 2023 there are 85 RMATs in the public domain. I also include FDA data on RMAT submissions via an FDA RMAT resource

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Live blogging Future of Genome Medicine: great talks by Feng Zhang & others

March 2, 2017 / Professor Paul Knoepfler, Ph.D.

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.

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In Depth Report from April 29th Paris Human Gene Editing Meeting

May 11, 2016 / Professor Paul Knoepfler, Ph.D.

Editor’s note. Caroline Simons attended both the April 28th (see her report on that here) and 29th Paris meetings on human gene editing/genetic modification. Today, we have her in depth report on the April 29th meeting. I have posted her piece in full with only minor edits. If you are in a rush you can

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Radical Vision for CIRM Future: Interview with New Prez Mills

December 5, 2014 / Professor Paul Knoepfler, Ph.D.

Change is in the air for the California stem cell agency, CIRM. It has a new President, Randy Mills, and CIRM is soon going to be like a new agency too. I recently did an interview with Mills that illuminates some of the big changes that are imminent. I also asked him what his favorite

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