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New molecular targets and proof-of-concept therapies for Autism Spectrum Disorders

Project description

Shrinking the autism research gap

Diagnosed before age 3 based on behavioural cues, autism is a major multifactorial neurodevelopmental disorder, affecting 1 in 100 child births worldwide. There is no cure and what is more, there is no one size fits all approach to autism management. The EU-funded THERAUTISM project will aim to shrink the autism research gap to expose the common molecular and cellular dysfunctions underlying autism-related behaviours. It will also investigate the possibility for a safe gene therapy vector – after it takes into account the properties of a new protein function to restore this new target and rescue social deficits in different preclinical models of autism. The aim is to validate new molecular therapeutic targets.

Objective

Autism is the major neurodevelopmental health public issue, affecting 1/100 child births worldwide. These disorders are diagnosed before the age of 3, based on behavioural cues: deficits in social interaction and communication as well as stereotyped and restrained behaviours. There is no medication to improve this condition. Most recent molecular targets identified within narrow frameworks (unspecific molecule, single tissue targeted, single disease model used) have failed in clinical trials. My first objective aims at thwarting this autism research gap, unravelling the common molecular and cellular dysfunctions underlying autism-related behaviours across several preclinical models and neuronal circuits. In particular, setting up translatomic analyses in these paradigms will identify and validate new molecular therapeutic targets. I recently deciphered one such molecular substrate, involving the loss of oxytocin transcripts in oxytocinergic axon terminals thus demonstrating the feasibility of this global approach. The second major objective of my project is to hijack the properties of a newly identified protein function to restore this new target and rescue social deficits in different preclinical models of autism. This would yield a novel and safe gene therapy vector which has never been explored before. Altogether, my research project will deliver strategic resources to the scientific and medical communities that will spur the development of new treatment options for autistic patients.

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Coordinator

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS
Net EU contribution
€ 1 499 025,00
Address
Rue michel ange 3
75794 Paris
France

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Links
Other funding
€ 0,00

Beneficiaries (1)