Gene therapy helps kids with immune disorder

	Tweet

A type of gene therapy helps restore a working immune system to children with a genetic disease called x-linked severe combine immune deficiency (SCID-X1), which raises the risk of serious infections, new research shows.

In fact, preventive medications, such as antibiotics, could be stopped for two of the four young children included in the study, Dr. Adrian J. Thrasher, at University College London, and his team report in The Lancet medical journal.

The therapy works by delivering a normal copy of gene that is mutated in kids with SCID-X1.

This approach helped restore various immune cells to normal or near-normal levels. At last follow-up, between 12 and 29 months after the treatment, “all patients were at home in normal family and social environments, without restriction on activities or exposure,” reports Thrasher’s team.

With refinements in technique, “there is potential to establish gene therapy as a standard therapeutic procedure for this type of disease,” the authors note.

In a related commentary, Drs. Marina Cavazzana-Calvo and Alain Fischer, from Hopital Necker-Enfants Malades in Paris, note that altogether, there have now been 18 cases of SCID that have been treated with this type of gene therapy, 17 of whom derived “clear and sustained clinical benefits.”

The current findings coupled with other promising reports “should encourage researchers to extend the treatment to target other life-threatening immunodeficiencies and, possibly, other genetic diseases” of the blood, the editorialists conclude.

SOURCE: The Lancet, December 18/25, 2004.

RELATED STORIES:
Gene therapy restores hearing in deaf animals   Autism gene identified by researchers at Yale working with a global research consortium   A Genetic Cause for Iron Deficiency   Gene Identified for Crohn’s Disease in Children   Prader-Willi syndrome: a form of obesity that can be explained   New mechanism in the regulation of human genes   Huntington’s Disease Deciphered   Feast, famine and the genetics of obesity: You can’t have it both ways   Developing Gene Therapy to Fight Blindness   New Evidence Ties Gene to Alzheimer’s   Newly-discovered mechanism can explain the Beckwith-Wiedemann syndrome   Gene Therapy Effective Treatment Against Gum Disease

Comments

[ + Post Your Own ]

Now you're in the public comment zone. What follows is not Armenian Medical Network's stuff; it comes from other people and we don't vouch for it. A reminder: By using this Web site you agree to accept our Terms of Service. Click here to read the Rules of Engagement.

There are no comments for this entry yet. [ + Comment here + ]