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Sickle cell anemia patient 'cured' by gene therapy, doctors say

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n a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Children's Hospital in Paris, researchers say.
People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood cells, causing blood to clog in the tiny vessels and organs of the body.

After 15 months since treatment, the patient -- who began therapy at age 13 -- no longer needs medication, and his blood cells show no further sign of the disease, according to a case report published Thursday in The New England Journal of Medicine.

"Since therapy was applied, he hasn't had any pain, any complications. He is free of any transfusions. He plays sports and goes to school," said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. "So we are quite pleased with the results." This success provides proof of concept for human patients, Leboulch said.

According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, "all the biological tests we perform lead us to think he is cured." Yet, she added, the answer to the question of whether he is truly cured "can be provided only by the longer follow-up."

Still, hopes are running high that patients with this very devastating disease can receive this therapy "in the next five years," Cavazzana said. "This is our hope, and we work very hard to attain it."


listen to the mad man
Even remission for a year would be an amazing quality of life boost for most sufferers. Outstanding news.


Is this true? I hope it is, I know a lot of kids with sickle cell and its heartbreaking losing people to it.


May I have a cookie?
Heard about this but haven't read into it much further. Any doctors/molecular biologists/geneticists on GAF care to comment?
Awesome. I have the sickle trait so I apparently have to be careful that a partner of mine doesn't also have it unless I want our kids to suffer from sicklecell. Glad breakthroughs are on their way.


Gonna be a long time before they try to use this on me.

I'm actually due to go to a conference about this method.

Awesome. I have the sickle trait so I apparently have to be careful that a partner of mine doesn't also have it unless I want our kids to suffer from sicklecell. Glad breakthroughs are on their way.

I'm glad your observant to that. In this day and age of science I find it absurd more couples don't get tested for it.


Professional Schmuck
We're going to cure EVERYTHING between biomechanical solutions, gene therapy, and actual DNA editing via CRISPR. What a wild, beautiful future.


Yeah, I read about this a few days ago. It's very relevant to me, considering I have the disease. While I'm kinda old to make full use of it, lots of younger people suffering from it will benefit from this treatment.
Hope it goes public soon, and at affordable prices. Most of the regions suffering from it are pretty poor.


Knowing nature, though... it'll create an entirely new genus of viruses. The fight never stops!
Even if it would, through modern technology humans would create altogether new biological threats to kill people. Homo homini lupus est


I know a lot of people are feeling pessimistic about the times we live in and what's to come, but I've never been more excited for the future advances in science and technology than I am now.


Does gene therapy work like it does in the movies?

They inject you with a virus and you're near death for a while?

no, based on the article they pretty much took the cells from their marrow, used targeted Chemo to destroy all cells then replaced the cells to propagate again.

This maybe a precursor to being able to add or delete certain genes.
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