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Deacetylase inhibitors for autism spectrum disorders an integrated preclinical...

Deacetylase inhibitors for autism spectrum disorders an integrated preclinical validation pipeline in brain organoids and in vivo models ASD is a very prevalent neurodevelopmental condition affecting more than 1% individuals, and represents a major unmet medical need since no effective treatment is available. Despite the phenotypic convergence of its core symptoms... ver más

31/12/2022

UMIL

150K€

Presupuesto del proyecto: 150K€

Líder del proyecto

UNIVERSITA DEGLI STUDI DI MILANO No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Ver 3 Participantes

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2022-12-31

ERC-2019-POC: ERC Proof of Concept Grant

I+D

Cerrada hace 6 años

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Información adicional privada

No hay información privada compartida para este proyecto. Habla con el coordinador.

3 Participantes

UMIL

108.35K€ | Lider

ELECTROLESS HARD COAT

317.97K€ | Participante

ISTITUTO EUROPEO DI ONCOLOGI...

41.66K€ | Participante

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Duración del proyecto: 30 meses Fecha Inicio: 2020-06-10
Fecha Fin: 2022-12-31

Líder del proyecto

UNIVERSITA DEGLI STUDI DI MILANO No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 150K€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto ASD is a very prevalent neurodevelopmental condition affecting more than 1% individuals, and represents a major unmet medical need since no effective treatment is available. Despite the phenotypic convergence of its core symptoms (impaired language, restriction in sociability and stereotypies, invariably coupled to anxiety and often associated to varying degrees of intellectual disability), ASD is genetically highly heterogeneous, with hundreds of bona fide causative genetic lesions. This genetic architecture breaks down ASD into a collection of rare and highly penetrant genetic syndromes, presenting key challenges but also decisive applicative opportunities: i) the accelerated regulatory path for drug approval/repurposing; and ii) the possibility that few paradigmatic syndromes may yield generalizable therapeutic inroads for ASD treatment. Duplication at 7q11.23 (7Dup), encompassing 26-28 genes, is one of best characterized ASD-causing copy number variations and offers unique translational opportunities, also because hemydeletion of the same interval causes Williams-Beuren syndrome, a condition characterized by hypersociability and language strengths, thereby providing a unique reference standard to validate treatments for the core ASD symptoms. Within my ERC-Consolidator project we successfully completed a high-throughput screening on 7q11.23 CNV patient-derived neurons and discovered that deacetylase inhibition lowers the abnormal levels of a key gene underlying the cognitive/behavioral traits of 7Dup. Having defined robust 7Dup neurodevelopmental and electrophysiological phenotypes, both in patients’ neuronal lineages and in mouse models, we now aim to generate proof of concept for the preclinical validation of selected deacetylase inhibitors in rescuing ASD phenotypes in vitro and in vivo. For this we pursue an integrated experimental and business plan, buttressed by key industrial partnerships, to advance the most inhibitors towards ASD treatment.

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