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Research Shows Way for Drug Therapy for Autism

Indian neuroscientists are part of an international team that has for the first time reversed symptoms of mental retardation and autism in mice, raising hopes of drug therapy for this disorder that affects one in 500 children.

Researchers at the National Centre for Biological Sciences (NCBS) and National Institute of Mental Health and Neuroscience (NIMHANS) in Bangalore have contributed to the breakthrough, reported this week's Proceedings of the National Academy of Sciences (US).

Scientists at the Massachusetts Institute of Technology (MIT) in the US and Seoul National University in South Korea are part of the team.

Autism is a developmental disorder characterised by varying degrees of deficiencies in communication skills and social interactions, along with restricted, repetitive and stereotyped patterns of behaviour.

A strain of mice, genetically engineered by the Nobel laureate Susumu Tonegawa and colleagues at MIT, were manipulated to model "fragile X syndrome (FXS)", the leading inherited cause of mental retardation and the most common genetic cause of autism.

"The condition, tied to a mutated gene in the X chromosome, causes mild learning disabilities to severe autism," researcher Sumantra Chatterji at NCBS told IANS. Presently, there is no effective treatment for FXS and other types of autism affecting all races and ethnic groups, he said.

"Our findings have identified a specific enzyme in the brain, called p21-activated kinase (PAK), as a potential target for drugs that may reverse many of the debilitating symptoms of FXS, and possibly autism, in children.

"The enzyme PAK affects the number, size and shape of connections between neurons in the brain," he said.

In the brain, these connections between neurons are formed by small protrusions called "spines", which are spread on branches of neurons called "dendrites". The numbers and shapes of dendrit
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