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Analysis: human immunity to Cas9 bigger #CRISPR therapeutic hurdle than off-targets?

April 6, 2018 / Professor Paul Knoepfler, Ph.D. / 1 Comment

Some in CRISPR-Cas9-land who are focused on potential future clinical applications are kind of rejoicing or at least sighing a breath of relief. This upbeat swing in the atmosphere (from investors especially) was sparked by retraction of that paper, the one initially reporting tons of supposed off-target CRISPR-Cas9 activity in mice, which turned out to be a “nothing […]

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Civil Lawsuits as a Public Health Strategy: Can Cases Brought by Injured Plaintiffs have a Broader Effect?

March 12, 2018 / Professor Paul Knoepfler, Ph.D. / 2 Comments

By Claire Horner In 2016, a law firm in California began looking for individuals who were “misled or harmed by stem cell treatment” in the southern California area (which was discussed on this blog here). While there had been other lawsuits filed by plaintiffs against individual clinics, this firm was looking to take a different

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Journal club: Jaenisch lab paper on epigenetic CRISPR-Cas9 rescue of Fragile X in a dish

February 26, 2018 / Professor Paul Knoepfler, Ph.D. / 3 Comments

Liu-et-al.-Figure-6C-Fragile-X-CRISPR-Cell-e1519673510685

There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat

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On President’s Day: Dealing with Reality Trump is Threat to Science & Democracy

February 19, 2018 / Professor Paul Knoepfler, Ph.D. / 8 Comments

On President’s Day, let’s think about our country’s leader, Donald Trump. Do we have to? Yeah, sometimes we do. For the first time in my lifetime, I see the President of the United States as a serious threat to overall science & to our country’s democracy. Trump is not well-acquainted with fact-based reality and that’s

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Why we shouldn’t view the human embryo as a gizmo even in the CRISPR era

January 31, 2018 / Professor Paul Knoepfler, Ph.D. / 4 Comments

My first job in science was as a lab technician at UCSD School of Medicine and a big part of that job was growing cells called HUVECs or human umbilical vein endothelial cells. We isolated and grew the HUVECs from umbilical cords that we retrieved from the maternity ward of the UCSD hospital, which first

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Is monkey cloning a breakthrough or a bad idea?

January 24, 2018 / Professor Paul Knoepfler, Ph.D. / 10 Comments

Is monkey cloning a good idea? We’re about to find out. A new Cell paper today reports the first cloning of monkeys via somatic cell nuclear transfer (SCNT), raising many questions. The paper from a team led by Qiang Sun is entitled, “Cloning of Macaque Monkeys by Somatic Cell Nuclear Transfer.” The highlight bullet points

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Some thoughts on taking risks in science & medicine

January 11, 2018 / Professor Paul Knoepfler, Ph.D. / 4 Comments

Are you a risk-taker or do you do everything possible to avoid risks? Taking risks in science is necessary, but is there a wise way to take the risks you do? Science and medicine need a certain amount of risk and risk-takers to make transformative leaps forward. The risks in biomedical science can be conceptual

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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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Keep calm & CRISPR on: perspectives on report of human Cas9 immunity

January 9, 2018 / Professor Paul Knoepfler, Ph.D. / 7 Comments

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

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Mesoblast gets FDA RMAT; List of 10 total designations so far

December 26, 2017 / Professor Paul Knoepfler, Ph.D. / 13 Comments

Australian stem cell biotech Mesoblast announced that it has received regenerative medicine advanced therapy (RMAT) designation from the U.S. FDA. This is very good news for the company and an encouraging development for the field. Interestingly, last month the FDA clarified that there is expanded RMAT designation that can include gene therapies too. At the Meeting

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