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Science!!!! Bitches
More Science! Using HIV to cure a disease!

'Bubble boy disease' cured using HIV with gene therapy at St. Jude's

Quote:They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies — HIV, the virus that causes AIDS.

A study out Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked.

"“The children are cured," Dr. Ewelina Mamcarz, one of the study leaders at St. Jude Children's Research Hospital in Memphis told NBC News. “They came to us as little infants, some of them as young as 2 months, with severe infections. Now they are home, living normal lives, attending daycare."

They don't require monthly injections, but it will take more time to see if it's a permanent fix, she said.

Omarion Jordan, who turns 1 later this month, had the therapy in December to treat severe combined immunodeficiency syndrome, or SCID.

"For a long time we didn't know what was wrong with him. He just kept getting these infections," said his mother, Kristin Simpson. Learning that he had SCID "was just heartbreaking ... I didn't know what was going to happen to him."

Omarion now has a normal immune system. "He's like a normal, healthy baby," Simpson said. "I think it's amazing."

Study results were published by the New England Journal of Medicine. The treatment was pioneered by a St. Jude doctor who recently died, Brian Sorrentino.

"This trial is his life's work," Mamcarz told NBC News.

SCID is caused by a genetic flaw that keeps the bone marrow from making effective versions of blood cells that comprise the immune system. It affects 1 in 200,000 newborns, almost exclusively males. Without treatment, it often kills in the first year or two of life.

"A simple infection like the common cold could be fatal," Mamcarz said.

The nickname "bubble boy disease" comes from a famous case in the 1970s — a Texas boy who lived for 12 years in a protective plastic bubble to isolate him from germs. A bone marrow transplant from a genetically matched sibling can cure SCID, but most people lack a suitable donor. Transplants also are medically risky — the Texas boy died after one.

Doctors think gene therapy could be a solution. It involves removing some of a patient's blood cells, using the modified HIV to insert the missing gene, and returning the cells through an IV. Before getting their cells back, patients are given a drug to destroy some of their marrow so the modified cells have more room to grow.

When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. The new therapy has safeguards to lower that risk.

A small study of older children suggested it was safe. The new study tried it in infants, and doctors are reporting on the first eight who were treated at St. Jude and at UCSF Benioff Children's Hospital San Francisco.

Within a few months, normal levels of healthy immune system cells developed in seven boys. The eighth needed a second dose of gene therapy but now is well, too. Six to 24 months after treatment, all eight are making all the cell types needed to fight infections, and some have successfully received vaccines to further boost their immunity to disease.

No serious or lasting side effects occurred.
(02-20-2016, 11:00 AM)Jason Wrote: Morty wept

(02-20-2016, 11:01 AM)LETTERS Wrote: Morty is Morty.
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I f*cking love AIDS!
(04-13-2018, 05:23 PM)r0cketfr0g Wrote: Ben, that was a pretty good post! Thanks!
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Another AIDS story for Ben!

Scientists say they found a cure for HIV in some mice. Humans could be next.

Quote:By Hannah Knowles, The Washington Post  updated at 12:48 PM

Researchers say they have removed HIV from the DNA of mice, an achievement the scientists say could be an early step toward an elusive cure for humans.

The breakthrough, detailed earlier this week in a study credited to more than 30 scientists from Temple University and the University of Nebraska Medical Center, was made possible by an antiviral drug in combination with the tool called CRISPR that can edit genes. The researchers eliminated HIV in nine of 23 mice that were modified so their immune systems better mimicked those of humans.  

Clinical trials for the gene-editing component of the cure could start as early as next year if the Food and Drug Administration approves them, said Kamel Khalili, one of the study’s senior investigators. But he and other HIV experts emphasized that there is a big scientific leap from promising results in mice to success in humans.

Clinical trials for the gene-editing component of the cure could start as early as next year if the Food and Drug Administration approves them, said Kamel Khalili, one of the study’s senior investigators. But he and other HIV experts emphasized that there is a big scientific leap from promising results in mice to success in humans.

“We knew what we needed to do, but the technology was unavailable,” Khalili told The Washington Post, saying he and his team had been awaiting a tool like CRISPR to combat a virus that “becomes part of the fabric of our chromosomes.” With gene editing finally a reality, he said, “the outcome was amazing.”

Once deadly, HIV can now be managed with a treatment called antiretroviral therapy. The therapy only keeps the virus in check; without constant medication, the virus will quickly decimate a patient’s ability to fight off sickness. HIV infects 37 million people worldwide, according to the latest data from the World Health Organization, and only about 22 million of those people receive antiretroviral therapy. Nearly 1 million people died of HIV-related issues in 2017, according to WHO.

Earlier this year, revelations that a second person had seemingly been rid of the virus raised hopes that another patient’s cure 12 years earlier was not a one-off victory. But scientists cautioned that it was too early to declare the anonymous second patient cured – and that, regardless, the case did not herald a widespread cure for the devastating condition. Both patients were treated with stem cell transplants, which experts say are risky, bring serious side-effects and would not be preferred for most patients.

Previously, Khalili’s team at Temple had found a way to remove significant amounts of HIV DNA from rats and mice. But the technique could not completely remove the infection. So Khalili’s lab joined forces with a University of Nebraska Medical Center lab attacking the problem in a different way. Together, the scientists combined the gene-editing strategy with a drug designed to beat back HIV.

Howard Gendelman from UNMC told The Post that his team’s experimental drug is engineered to act over a longer time than normal therapies, meaning it can be administered every couple of months instead of every day. It is also better able to target HIV in the body, he said. It is crucial that gene editing remove every last bit of HIV, he said, and the drug makes that task easier.

“If you can reduce the amount of virus that’s left for CRISPR, the likelihood that the CRISPR will be effective will go up enormously,” he said.

But if Khalili’s team is able to move forward with trials with humans, it will use standard drugs rather than the one developed by UNMC’s lab, since it probably would not be approved yet for use, Khalili said.

Steven Deeks, a professor at the University of California at San Francisco who has worked extensively on HIV, said the use of gene editing to remove HIV from a live animal is a notable step forward.

But he cautioned that using the technique on humans will be far more challenging: Scientists will have to grapple with more variations in the virus, more difficulties in delivering the gene-editing technology and the possibility of cutting up human genes while trying to target HIV, he said. Those are formidable problems, Deeks said, especially with success depending on removing the virus completely.

“For this approach to work, they have to really knock out 100 percent of the genomes – you can’t leave anything behind,” Deeks said. “One of them can reignite the whole process.”

The scientists behind the new study recognize those challenges. Khalili said researchers are in the process of publishing another study on the use of gene editing to target HIV in primates and are hopeful about bringing the solution to human subjects. But the scientists need to make sure their techniques are safe, he said. They also want to increase their methods’ chances of working from the roughly one-third success rate they saw with mice.

Gendelman said that while modern drugs are good at keeping HIV at bay, a permanent cure would bring big benefits to patients. Constant drug treatment can contribute to other health complications, he said, and just keeping the pills in your medicine cabinet can invite unwanted scrutiny from family or significant others.

“There’s a tremendous stigma,” he said. “Every time you take those pills you’re reminded that you have HIV.”
(02-20-2016, 11:00 AM)Jason Wrote: Morty wept

(02-20-2016, 11:01 AM)LETTERS Wrote: Morty is Morty.
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They're going to find a cure for humans in mice?
(04-13-2018, 05:23 PM)r0cketfr0g Wrote: Ben, that was a pretty good post! Thanks!
Switch Friend Code: SW-3450-8782-7709
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(07-08-2019, 04:32 PM)Ben Wrote: They're going to find a cure for humans in mice?

Alexa, what was the Black Plague?
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