Scientists Spot Possible Treatment for Rett Syndrome

Rare disorder afflicts girls and involves neurological, cardiac and respiratory woes

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TUESDAY, Feb. 10, 2009 (HealthDay News) -- Researchers may have found a new treatment for Rett syndrome, a rare inherited disease in girls that brings on autism-like symptoms along with heart and breathing complications.

A team from the Whitehead Institute for Biomedical Research and MIT conducted tests involving a mouse model of Rett syndrome. They found that daily shots of the active fragment from a protein called insulin-like growth factor-1 (IGF-1) helped with movement and breathing irregularities.

"This is the first realistic way for a drug-like molecule injected into the bloodstream to relieve Rett syndrome symptoms," Whitehead researcher Rudolf Jaenisch said in a news release issued by the institute.

"Although the treated mice get better and their symptoms don't progress as fast as they normally would, the treated mice still get the symptoms. So it's definitely not a cure, but it could be a co-therapy," study researcher Emanuela Giacometti said the same news release.

Most children with Rett syndrome, an inherited neurological disease, have a non-functioning MeCP2 gene which may prevents nerve cells from maturing and forming the internal network necessary for the body's systems to communicate with itself. Previous studies in mice found that turning on MeCP2 helped the rodents resume normal body movement and lifespan.

The study's team investigated IGF-1 as a treatment based on its previous work that had shown that high levels of the protein in the brain invigorate the growth of the connections between nerve cells, called synapses. This growth helps complete the network that is essential for brain function, they explained.

The study appears in this week's issue of the Proceedings of the National Academies of Sciences.

More information

The Rett Syndrome Research Foundation has more about Rett Syndrome.

SOURCE: Whitehead Institute for Biomedical Research, news release, Feb. 9, 2009


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