Rett Syndrome treatment for mice developed

CAMBRIDGE, Mass., Feb. 1 (UPI) -- Scientists at Whitehead Institute for Biomedical Research in Cambridge, Mass., report reducing some symptoms of Rett Syndrome in mice.

Rett Syndrome is a neurological disorder occurring primarily in girls. While some existing therapies might ease particular symptoms of the condition, there's no current way to address the syndrome at a molecular level.

Whitehead researchers, in collaboration with scientists at Brandeis University, report dramatically reducing certain manifestations of Rett Syndrome in mice, possibly leading to the development of therapies for people.

"This is the first time we've successfully reduced the awful symptoms of Rett Syndrome using transgenic techniques," said Whitehead biologist Rudolf Jaenisch, senior author of the paper. "Once we understand the molecular mechanisms of the disease we may be able to design rational strategies that may eventually be useful ... in people."

Rett Syndrome occurs in about 1 in 15,000 people and is caused by a defective gene on the X chromosome. Most boys with Rett Syndrome die before birth; girls develop normally until about six to 18 months, then show symptoms such as loss of speech, loss of voluntary motor control and seizures.

The research appears in the Feb. 2 issue of the journal Neuron.