Gene therapy relieves sickle cell in world first

2017-03-02 20:00
(iStock)

(iStock)

Multimedia   ·   User Galleries   ·   News in Pictures Send us your pictures  ·  Send us your stories

Paris - Scientists have used gene therapy to relieve the symptoms of a teenager suffering from sickle cell disease (SCD) in a world-first breakthrough, they say.

SCD is an inherited disease caused by a gene mutation that results in red blood cells losing their usual doughnut-like appearance and taking on a sickle or crescent moon shape.

Sufferers - about five million worldwide - often have anaemia and get tired easily, run a higher risk of infections and stroke and experience bouts of severe body pain.

Many need chronic blood transfusions.

But a team from the AP-HP university hospital group in Paris, the Imagine Institute of Genetic Diseases and gene therapy company bluebird bio said they managed to get a teenager off transfusions.

After treatment

The boy was the first person to be treated in Paris in October 2014, for sickle cell disease in a clinical trial with gene therapy. Others have been tested since, but no official results published.

The team collected so-called haematopoietic stem cells, which give rise to red blood cells, from the bone marrow of the youngster, then aged 13.

The immature cells were treated with a therapeutic gene, carried in a de-activated virus, which recoded their DNA to correct blood cell production.

The treated cells were then re-injected into the boy's body.

Thursday's results, published in the New England Journal of Medicine, report on the teen's health 15 months after treatment.

He was still doing well after this point, but an official, updated status has yet to be published in a peer-reviewed journal.

"He is well, he no longer needs monthly (blood) transfusions, anti-pain medication, or hospitalisation," said study leader Marina Cavazzana.

SCD is common in Africa, where up to 40% of a country's population can carry the mutated gene, though most never get sick.

Diagnosis

French researchers last month reported progress in developing a rapid, on-the-spot diagnosis for the disease.

Early results from a trial in Togo, Mali and the Democratic Republic of Congo suggested the Sickle Scan was a faster, cheaper blood test than existing ones relying on lab equipment, its makers said.

Rapid diagnosis is crucial to start SCD sufferers, especially young children, on potentially life-saving treatment.

Read more on:    france  |  health  |  medicine

Join the conversation!

24.com encourages commentary submitted via MyNews24. Contributions of 200 words or more will be considered for publication.

We reserve editorial discretion to decide what will be published.
Read our comments policy for guidelines on contributions.
NEXT ON NEWS24X

Inside News24

 
/News
 

6 myths about male cancer

It is important to be aware of the most prevalent cancer diseases amongst men in our country.

 
 

You won't want to miss...

Who are the highest paid models of 2017?
10 gorgeous plus-sized models who aren't Ashley Graham
5 top leg exercises for men
10 best dressed men of 2017
Traffic Alerts
Traffic
There are new stories on the homepage. Click here to see them.
 
English
Afrikaans
isiZulu

Hello 

Create Profile

Creating your profile will enable you to submit photos and stories to get published on News24.


Please provide a username for your profile page:

This username must be unique, cannot be edited and will be used in the URL to your profile page across the entire 24.com network.

Settings

Location Settings

News24 allows you to edit the display of certain components based on a location. If you wish to personalise the page based on your preferences, please select a location for each component and click "Submit" in order for the changes to take affect.




Facebook Sign-In

Hi News addict,

Join the News24 Community to be involved in breaking the news.

Log in with Facebook to comment and personalise news, weather and listings.