Home / Software & Service News / Gene therapy gives ‘bubble babies’ immune systems

Gene therapy gives ‘bubble babies’ immune systems

Initial results from a new gene therapy technique suggest it could open the doors to a cure for “bubble baby” disease. Lacking the ability to ward off even the most common infections, infants born with the genetic disorder — known as severe combined immunodeficiency (SCID) — usually die before their second birthday. And, those untreated must be kept in isolation from the outside world, hence the term “bubble baby.” Even with the best available treatment (a stem cell transplant), around 30 percent of children end up dying by the age of 10.

Roughly four months after the genetic modifications, six out of seven babies are out of protective isolation and leading healthy lives, according to doctors at St Jude Children’s Research Hospital. The remaining infant’s immune system is still in the process of constructing itself.

The patients in the study were all born with the inherited X-linked SCID, which is limited to boys as it’s triggered by a genetic defect in the male X chromosome. The treatment they received uses an inactivated form of HIV to apply genetic modifications to bone marrow — which is prepped using low doses of chemotherapy — in order to kickstart it to produce all three major immune cell types. “The initial results also suggest our approach is fundamentally safer than previous attempts,” said lead study author Dr. Ewelina Mamcarz.

At first glance, the treatment is being viewed as a possible cure. But, more work is needed — specifically, the babies need to be monitored to ensure they remain stable with no side effects. Their response to vaccination will also need to be tracked.

Source: St. Jude Children’s Research Hospital

Click Here For Original Source Of The Article

About Ms. A. C. Kennedy

Ms. A. C. Kennedy
My name is Ms A C Kennedy and I am a Health practitioner and Consultant by day and a serial blogger by night. I luv family, life and learning new things. I especially luv learning how to improve my business. I also luv helping and sharing my information with others. Don't forget to ask me anything!

Check Also

Get a full surround-sound speaker set for more than 40% off

TwitterFacebook

Heads up: All products featured here are selected by Mashable's commerce team and meet our rigorous standards for awesomeness. If you buy something, Mashable may earn an affiliate commission.

Speakers have come a long way since their invention in the late 1800s. Today, they're even included on everything from smartphones, to computers, to television sets — but not always with the best quality. Luckily, if you're ready to take your sound system up a notch, you'll find no shortage of options. Speakers come in all shapes and sizes: some of them wireless, some waterproof, and some that even tell you jokesRead more...

More about Bluetooth, Speakers, Bluetooth Speakers, Speaker, and Bluetooth Speaker

css.php