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Ottawa Study Reverses Autism Symptoms in Mice, Eyes Drug Path

Ottawa researchers have reversed autism-related behaviours in adult mice by targeting a receptor on endothelial cells. The team, based at The Ottawa Hospital and the University of Ottawa, has filed a patent application and plans further experiments.

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Ottawa Study Reverses Autism Symptoms in Mice, Eyes Drug Path
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A research team led from Ottawa has reversed autism-related behavioural symptoms in adult mice, and it says the finding points to a new target for future treatments. The work comes from The Ottawa Hospital and the University of Ottawa.

What the study found

The research was led by Baptiste Lacoste, a senior scientist at The Ottawa Hospital and a professor at the University of Ottawa. Julie Ouellette, a former PhD student of Lacoste's, headed the research team.

The results were published in the journal Neuron under the title "Purinergic receptor activation rectifies autism-associated endothelial dysfunction."

The team worked with a mouse model that carries the 16p11.2 deletion, described as one of the most common genetic mutations seen in autism. In these mice, endothelial cells had half the normal level of ATP.

The researchers then activated a receptor called P2Y2, and that restored the cells' function. Lacoste put it this way: "It's as if these cells are asleep, and now we can wake them up."

Symptoms reversed in adult mice

The treatment reversed behavioural symptoms in adult mice. Those included hyperactivity, repetitive movements and motor learning impairment.

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The drug used in the experiments activates P2Y2. It is already approved for humans in Japan and South Korea to treat dry eye syndrome. That is a separate use from anything tested in this study, and the work so far has been in mice only.

What happens next

The team has filed a patent application for using P2Y2 activation in the blood vessels to treat autism symptoms.

The next experiment is already planned. The researchers intend to treat mice earlier in life to see whether early treatment has additional benefits.

The sources do not say when, or whether, a human trial might follow. No timeline for testing in people has been announced, and nothing in the published results shows that the same effect would appear in humans. For now, this is a laboratory finding in a genetic mouse model.

Why it matters to Ottawa readers

The study was led by scientists at two of the city's major research institutions, The Ottawa Hospital and the University of Ottawa. Families in the region who follow autism research may want to know that work with Ottawa roots is looking at a different kind of target than is usually discussed.

Anyone with questions about autism care or treatment today should speak with their own health care provider. Nothing in this research changes current care, and no new treatment is available as a result of it.

Sources: GEN (Genetic Engineering & Biotechnology News), Education News Canada (University of Ottawa release)

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