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ASTRO-EDITING

Financiado

Cerrado

Targeting ASTROcytes with cutting edge EDITING technologies to treat the Alexand...

Targeting ASTROcytes with cutting edge EDITING technologies to treat the Alexander disease Alexander disease (AxD) is an autosomal dominant neurodegenerative disorder caused by missense mutations in the gene encoding the glial fibrillary acidic protein (GFAP), the major intermediate filament protein in astrocytes. Accum... ver más

30/06/2022

OSR

183K€

Presupuesto del proyecto: 183K€

Líder del proyecto

OSPEDALE SAN RAFFAELE SRL No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2022-06-30

MSCA-IF-2019: Individual Fellowships

I+D

Cerrada hace 5 años

0% 100% 100%

Información adicional privada

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

1 Participantes

OSR

183.47K€ | Lider

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Duración del proyecto: 27 meses Fecha Inicio: 2020-03-27
Fecha Fin: 2022-06-30

Líder del proyecto

OSPEDALE SAN RAFFAELE SRL No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 183K€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Alexander disease (AxD) is an autosomal dominant neurodegenerative disorder caused by missense mutations in the gene encoding the glial fibrillary acidic protein (GFAP), the major intermediate filament protein in astrocytes. Accumulation of GFAP aggregates in Rosenthal fibres leads to impairment of proteasomal activity and hyperactivation of the stress response, thus compromising astrocyte functions and altering the homeostasis of the central nervous system (CNS). Currently, this orphan disease lacks a cure. The proposed project aims at providing in vivo proof-of-concept of safety, efficiency and efficacy of a novel and definitive AAV gene therapy approach based on gene editing / base editing strategies targeting GFAP mutational hotspots to recover pathological phenotypes in astrocytes. The candidate expects to outline novel editing platforms for widespread in vivo targeting of CNS astrocytes that could be applied for the treatment of AxD, as proposed in ASTRO-EDITING, and prospectively for disease modelling studies and therapeutic treatments of other neurological and psychiatric disorders by targeting pathological pathways involved in primary astrocyte degeneration or dysfunctional/maladaptive astrogliosis. The proposal addresses the European issue of Horizon 2020, in the social challenge Health, Demographic Change and Wellbeing endeavours for the call Better health and care, economic growth and sustainable health disease, subject SC1-BMC-04-2018 Rare Disease European Joint Programme Cofund. The expertise of the host laboratory, the international environment of OSR and the personalized career development plan will significantly reinforce candidate's competences and knowledge in the gene therapy field, expand his portfolio of therapeutic areas, create a new research niche, and strengthen his organizational, management and interpersonal skills, thus favouring the progressive acquisition of independence as experienced researcher in the scientific community.

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