gene therapy

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

James Wilson, Gene therapy

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

bluebird bio, sickle cell gene therapy

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

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

FOGM-venue-Scripps-Seaside-Forum

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

Keep-calm-CRISPR-on

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

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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