gene therapy Archives

FDA OKs risky, pioneering OSK rejuvenation trial with Sinclair’s ER-100

February 11, 2026 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

The FDA has cleared a trial of ER-100 from Life Biosciences for eye rejuvenation. In the trial, ER-100 will deliver inducible expression of the three reprogramming factors. OCT4, SOX2, and KLF4 are colloquially known together as OSK. This trial is an extension of the work of Harvard professor and longevity enthusiast David Sinclair. The hypothesis here […]

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Weekly reads: Sarepta & BrainStorm Cell woes, peptide craze, FDA AI hallucinating

July 27, 2025 / Professor Paul Knoepfler, Ph.D. / Leave a Comment

What is the deal with Sarepta lately? Things are spiraling. Before we dig into that, let’s give a RIP to Ozzy Osbourne, who had struggled with health issues. He was one of dozens of celebs to turn to unproven stem cells. Also, a heads up that student and stem cell researcher Parmin Sedigh and I

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2nd chances after big misconduct? He Jiankui, James Wilson, others

December 8, 2022 / Professor Paul Knoepfler, Ph.D. / 3 Comments

There have been rare instances like with scientist James Wilson where researchers were involved in what I would call major misconduct but somehow managed to bounce back. In a sense, they were given second chances in part by regulators but also by other scientists or supporters. However, after extraordinary misconduct, especially contributing to the death

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Bluebird bio sickle cell gene therapy trial halted on cancer worry

February 16, 2021 / Professor Paul Knoepfler, Ph.D. / 2 Comments

Recently I was going through a list I’m updating of regenerative medicine biotech companies including Bluebird bio. Now just a few days later the world seems turned upside down for the firm and maybe to some degree for other companies doing related work. Bluebird bio blues: 2 trial participants get cancer News broke today that

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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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Extraordinary epidermis regeneration in child via combo stem cell-gene therapy

November 8, 2017 / Professor Paul Knoepfler, Ph.D. / 3 Comments

Hirsch-et-al-epidermal-regeneration-Fig.-2a

Epidermis regeneration is an important approach to helping patients with a variety of serious skin problems that can arise due to genetic conditions or injuries such as severe burns. An exciting new Nature paper reports epidermis regeneration on a strikingly large-scale. The child who is the subject of the study suffers from a sometimes fatal genetic

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