Tuesday, April 23, 2019

HIV as a way to cure other diseases???


I was astonished to read this report.

US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or "bubble boy" disease.

. . .

The babies, born with little to no immune protection, now have fully functional immune systems.

Untreated babies with this disorder have to live in completely sterile conditions and tend to die as infants.

The gene therapy involved collecting the babies' bone marrow and correcting the genetic defect in their DNA soon after their birth.

The "correct" gene - used to fix the defect - was inserted into an altered version of one of HIV, the virus that causes AIDS.

Researchers said most of the babies were discharged from the hospital within one month.

Dr Ewelina Mamcarz of St Jude, an author of the study, said in a statement: "These patients are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives."

There's more at the link.

SCID got its nickname of "bubble boy disease" from the tragic case of David Vetter, who had to live in a plastic "bubble" tent facility from birth until he died.





SCID used to be a death sentence.  To think that HIV, one of the most ravaging, most lethal viruses in recent memory can be gene-edited to be not only harmless, but actually medically helpful in this way, is mind-boggling.  Kudos to all the researchers and staff involved.

Peter

7 comments:

Orvan Taurus said...

Amazing what people can get up to when allowed to think for real.
This creature is mightily impressed indeed.
Taking one of Nature's own swords and saying, "Hey, plowshare!"

Jeff Weimer said...

Not just other diseases, an autoimmune disease.

D.J. said...

They've tried it with Lymphoma as well.

https://xkcd.com/938/

Technomad said...

Wonderful news!

Beans said...

One autoimmune disease to rule them all,
One autoimmune disease to find them.
One autoimmune disease to bring them all, and in the body bind them...

Eric Wilner said...

I figured, many years ago, that HIV would eventually be modified for treating allergies.
I'm very surprised, though, to hear of it being used to treat immunodeficiency.
Truly wondrous good news!

TheOtherSean said...

Minor tangent: The story in the "Boy in the Bubble" movie was a composite of the stories of David Vetter (SCID) and that of Ted DeVita (aplastic anemia).