from the technology-on-the-bleeding-edge dept.
A gene therapy trial has shown success in reducing bleeding episodes caused by the inherited blood disorder hemophilia B. Ten participants received a virus that stimulated the production of the blood-clotting protein factor IX. Nine have had no bleeding episodes, while eight no longer need the gene therapy injections every few days:
Previous gene therapy trials for hemophilia B didn't go well, either because patients' immune systems destroyed the modified cells or the cells didn't make enough factor IX. In the new trial, sponsored by Spark Therapeutics and Pfizer, researchers gave the patients' liver cells the gene for an unusually potent version of the factor IX protein. That allowed the team to lower the vector dose, minimizing immune responses. Two patients had elevated liver enzymes in reaction to the vector, but those levels came down after they received steroids. Only 20% of hemophilia patients have the B form, but efforts are also underway to use gene therapy to treat the most common type, hemophilia A.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant (DOI: 10.1056/NEJMoa1708538) (DX)
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Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures. "I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.
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Previously: Gene Therapy Cure for Sickle-Cell Disease
Gene Therapy to Kill Cancer Moves a Step Closer to Market
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FDA Approves a Gene Therapy for the First Time
Gene Editing Without CRISPR -- Private Equity Raises $127 Million
FDA Committee Endorses Gene Therapy for a Form of Childhood Blindness
FDA Approves Gene Therapy for Non-Hodgkin's Lymphoma
Gene Therapy and Skin Grafting for Junctional Epidermolysis Bullosa
Gene Therapy for Spinal Muscular Atrophy Type 1
Biohackers Disregard FDA Warning on DIY Gene Therapy
CRISPR Used to Epigenetically Treat Diseases in Mice
Gene Therapy Showing Promise for Hemophilia B
Gene Therapy for Retinal Dystrophy Approved by the FDA
CRISPR Treatment for Some Inherited Forms of Lou Gehrig's Disease Tested in Mice
(Score: 2) by Runaway1956 on Monday December 11 2017, @06:59PM (2 children)
Our bleeder has some kind of blessed/cursed thing going on. He'll bleed with little or no reason - he's been shot, stabbed, slashed, car wrecks, trees fallen on him. No one knows HOW in hell he's lived into his seventies. A freaking BLEEDER, FFS, with more trauma behind him than most non-bleeders will ever survive. Go figure . . . he has to have a patron saint or something.
(Score: 2) by Snow on Monday December 11 2017, @09:23PM
When I was in elementary school, I would start bleeding from my nose, just like Eleven does on Stranger Things.
I didn't have any magical powers though. Just a nose picking problem.
(Score: 0) by Anonymous Coward on Monday December 11 2017, @09:53PM
You bleed out faster if your body is pumping faster, so that could be part of it.
If you don't have any brain defects that result in a brain bleed or aneurism, having blood that doesn't clot as much could be beneficial, since it can avoid clotting related plaque buildup in your veins and arteries that might otherwise have the opportunity to break off or cause scarring.
Another one that helps with longevity is not having any of the genes that can cause arterial hardening, which often leads to tears that can cause internal bleeding or aneurisms.