Gene therapy works for bubble boy disease

Gene therapy works for bubble boy disease

According to the NEJM paper, patients first received bone marrow ablation with busulfan in order to clear up space for the gene therapy.

All of the children are now producing the immune cells needed to fend off the barrage of germs that humans encounter in their everyday lives, according to the study, published Wednesday (April 17) in the The New England Journal of Medicine. An author of the study one Dr Ewelina Mamcarz 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".

These new findings are a step forward in the current gene replacement therapy, the earlier versions of which has been known to be less efficient procedure that puts the patients at a high risk of leukaemia. "Now they are home, living normal lives, attending daycare".

Flames exit playoffs after loss in Game 5 to Colorado
The 37-year-old Smith took a skate to his leg when Avalanche forward Colin Wilson slid into him in the first period. The Colorado Avalanche look to clinch the Western Conference first round over the Calgary Flames in game five.

Bucks take Game 2
Based on how the Bucks were defending early, the Pistons have to work on their outside shooting in the next few days. He played limited minutes against Memphis on April 9 and sat out the regular-season finale against NY the next night.

Blackpool Easter bank holiday weekend weather: Temperatures set to soar
Most people will get a chance to enjoy the good weather but the warmest conditions are expected in the southeast of England. To put things into contrast, Bude was the warmest place in the country on Easter Sunday previous year - at a meagre 11.6C.

The exact incidence of X-SCID is unclear, according to the National Institutes of Health, but it likely occurs in 1-in-50,000 to 100,000 newborns, much more frequently in males than in females. Currently, the best treatment for SCID-X1 is bone marrow transplantation with a tissue-matched sibling donor.

Shares of a small biotech company went stratospheric this week following news that its gene therapy had cured several children with a rare immune-deficiency disease. However, most patients did not have reconstitution of functional B cells, and their T-cell immunity waned over time, meaning they had to receive additional therapies such as bone marrow transplantation and gene therapy. The team performed a dual-center, Phase I-II safety and efficacy study of a lentiviral vector to transfer IL2RG cDNA to bone marrow stem cells in the infants with newly diagnosed SCID-X1. A study out 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.

One concern with gene therapy is that, after inserting a gene into people's DNA, genes that are next to the insertion site may turn cancerous, as happened in prior cases where people developed leukemia.

Related Articles