Search Results for: sickle cell

Weekly reads: FDA OKs 2 gene therapies for sickle cell, new Paolo Macchiarini series, Fujifilm

Marie Tornyenu, Casgevy gene therapies for sickle cell

The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for […]

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Weekly reads: CRISPR sickle cell, Parkinson’s, pig-human chimera concerns

CRISPR gene editing

CRISPR gene editing has made rapid progress heading from bench to bedside. Perhaps the fastest has been its progress toward clinical use to combat sickle cell disease. We’ll start with a new paper on one major effort here. CRISPR gene editing. This process often involves cutting DNA, which then can be used as an opening to

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Weekly reads: reprogramming hearing loss, heart disease, eye drops, sickle cell

Regener-Eyes, eye drops

It’s mostly been a week of good and encouraging news in the regenerative medicine space including with gene therapies maybe with the exception of some eye drops warnings (more below). There’s realistic hope for an approved sickle cell disease soon. I also see some long-term positive news on hearing loss research. Hematopoietic stem cell transplant

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Weekly reads: stem cell good news, bad news on sickle cell, MS, IPSCs

victoria gray family crispr sickle cell b

Let’s start with some stem cell good news on evolving approaches to treat sickle cell disease involving stem cells. Then on another related front things were more concerning. Stem cell good news on sickle cell disease, a complication Sickle Cell Disease News reports Adding Briquilimab Boosts Success of Sickle Cell Stem Cell Transplant. This is a

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Weekly reads: death in CRISPR trial, sickle cell, nose picking & dementia, epilepsy

CRISPR gene editing

As both a scientist who works on stem cells, cancer, and CRISPR, and a research advocate I’ve been fortunate to meet many patient advocates over the years. Some have been participants in clinical trials themselves. Benefits & risks of clinical trial participation It is very sobering to find out that a clinical trial participant has

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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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Recommended reads: CRISPR for Sickle Cell, Parkinson’s, & more

victoria gray family crispr sickle cell b

I’m playing catch-up on some reading given how busy I’ve been and this includes a groundbreaking NEJM pub on CRISPR for Sickle Cell and Thalassemia. CRISPR for Sickle Cell From December, here’s the key paper in the NEJM: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. There’s a lot to like about this clinical

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Weekly reads: George Church’s stem cell startup GC Therapeutics, coral for knees, blast from the past

Parastoo Khoshakhlagh, Alex Ng, GC Therapeutics

It’s always fun to see startups with cool ideas in the regenerative medicine space and a new one called GC Therapeutics got some nice coverage this week. What do you think the name of this firm stands for? I have one main idea I mention below. Before we jump into it, I want to introduce

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Weekly reads: Vertex stem cells for diabetes, storing iPS cells on the Moon, science hype award nominees

Felicia Pagliuca, stem cells for diabetes

How is research looking on stem cells for diabetes? I’m feeling more encouraged about the clinical research in that area. Before we jump into that let’s talk about two other things.  The big news of the week was the felony plea deal by Liveyon’s John Kosolcharoen to charges related to marketing an unapproved cell therapy

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