---
title: "Scientists Gene-Edited HIV to Cure “Bubble Boy” Disease"
description: "Researchers say they've cured 10 infants born with SCID-X1, also known as \"bubble boy\" disease, using an HIV-assisted gene therapy."
date: "2019-04-18"
modified: "2019-04-18"
authors:
  - name: "Kristin Houser"
    link: "https://futurism.com/authors/kristin"
url: "https://futurism.com/neoscope/scientists-hiv-cure-bubble-boy-disease"
categories:
  - "Health & Medicine"
tags:
  - "gene therapy"
  - "HIV"
  - "scid-x1"
  - "the digest"
---

# Scientists Gene-Edited HIV to Cure “Bubble Boy” Disease

![Researchers say they've cured 10 infants born with SCID-X1, also known as "bubble boy" disease, using an HIV-assisted gene therapy.](<https://futurism.com/wp-content/uploads/2019/04/scientists-hiv-cure-bubble-boy-disease1.png>)
*\<em\>Image: Darwin Laganzon via Pixabay/Tag Hartman-Simkins\</em\>*

## Normal Lives

Researchers from St. Jude Children’s Research Hospital have reportedly [used HIV to cure infants](<https://www.stjude.org/media-resources/news-releases/2019-medicine-science-news/st-jude-gene-therapy-cures-babies-with-bubble-boy-disease.html>) born with "bubble boy" disease — a remarkable example of hijacking a deadly virus for a new treatment.

"The children are cured," researcher Ewelina Mamcarz [told *NBC News*](<https://www.nbcnews.com/health/kids-health/bubble-boy-disease-cured-using-hiv-gene-therapy-st-jude-n995631>). "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."

## Danger Everywhere

X-linked severe combined immunodeficiency (SCID-X1) is a rare genetic disorder caused by a mutation in the interleukin-2 receptor subunit gamma (IL2RG) gene.

People born with SCID-X1 lack a functioning immune system, which makes exposure to any germ potentially life-threatening. In fact, many children born with SCID-X1 die [before their second birthday](<https://apnews.com/38abc495ee3840289d59ac35e0d21af3>).

## Gene Therapy

In a [study](<https://www.nejm.org/doi/full/10.1056/NEJMoa1815408>) published Thursday in the *New England Journal of Medicine*, the St. Jude's team details how it developed a [gene therapy](<https://futurism.com/neoscope/gene-therapy-deafness-mice>) to effectively cure 10 infants born with SCID-X1.

After collecting bone marrow samples from each patient, they used a modified version of HIV to place a non-mutated IL2RG gene into the patients' stem cells. Then they dosed the patients with busulfan, a drug that helps make room for donor stem cells to grow, and returned the cells to the patients' bodies.

Most were discharged from the hospital within a month.

"While longer follow-up is needed to assess any late effects of treatment," researcher Mort Cowan said in a [news release](<https://www.stjude.org/media-resources/news-releases/2019-medicine-science-news/st-jude-gene-therapy-cures-babies-with-bubble-boy-disease.html>), "these results suggest most patients treated with this gene therapy will develop a complete durable immune response without side effects."

**READ MORE:** [St. Jude gene therapy cures babies with 'bubble boy' disease](<https://www.stjude.org/media-resources/news-releases/2019-medicine-science-news/st-jude-gene-therapy-cures-babies-with-bubble-boy-disease.html>) \[St. Jude\]

**More on gene therapy:** *[Scientists Used Gene Therapy to Cure Deafness in Mice](<https://futurism.com/neoscope/gene-therapy-deafness-mice>)*

### Author social links  
[LinkedIn](<https://www.linkedin.com/in/kristinjhouser>)