gene therapy

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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