Could a simple protein jab treat dwarfism?

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Press Trust of India London
Last Updated : Sep 24 2013 | 4:46 PM IST
A simple protein jab that could reverse the effects of dwarfism in children may be available within three to five years, scientists say.
In a breakthrough study, French scientists injected a protein molecule in mice with the condition achondroplasia - a genetic mutation that causes the vast majority of human cases of dwarfism.
Two protein injections were given every week for three weeks to the mice, which allowed them to grow fully and the painful complications of their condition were also removed.
Since three weeks is roughly equivalent to 15 human years, according to researchers, a similar treatment could theoretically work on children with achondroplasia, up to puberty, and without the need for daily injections.
However, adults with achondroplasia, who have already gone through puberty, would not be able to benefit from the treatment.
"I'm very excited by this. The treatment is so simple - it's just the injection of a protein, and the results show it really helped," said Elvire Gouze, a researcher at the Mediterranean Centre for Molecular Research in the southern French city of Nice.
"This is a first, and although there are more steps we need to take before trials on humans, nothing so far has indicated it couldn't also work for people with dwarfism," she told 'The Local'.
Gouze believes that researchers could be ready within three to five years to treat humans with this technique.
She added that the molecule injection also relieved conditions associated with dwarfism.
"It didn't just help with height, but it also made a difference with all the other complications," she said.
Humans with dwarfism often suffer back, lumbar and cervical pain, as well as muscular weakness and respiratory problems.
The study is published in the journal Science Translational Medicine.
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First Published: Sep 24 2013 | 4:46 PM IST

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