Hola,
¿eres nuevo aquí?

Regístrate gratis y conecta tu empresa con financiación pública, partners y proyectos.

Tengo cuenta

Regístrate

Ver video

muscleTDP

Financiado

The potential contribution of skeletal muscle to TDP-43 associated ALS.

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by motor neuron degeneration that leads to progressive muscle atrophy. Currently only a few FDA-approved drugs are available for ALS, which me... ver más

31/05/2026

STICHTING RADBOUD...

203K€

Presupuesto del proyecto: 203K€

Líder del proyecto

STICHTING RADBOUD UNIVERSITEIT No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2024-03-12

HORIZON-MSCA-2023-PF-01-01: MSCA Postdoctoral Fellowships 2023 ExpectedOutcome:Project results are expected to contribute to the following outcomes:

I+D

Cerrada hace 1 año

0% 100% 100%

1 Participantes

STICHTING RADBOUD UNIVERSITE...

203.46K€ | Lider

Conecta tu I+D

¿Tienes un proyecto y buscas un partner? Gracias a nuestro motor inteligente podemos recomendarte los mejores socios y ponerte en contacto con ellos. Te lo explicamos en este video

Proyectos interesantes

FJC2021-046836-I Studying the central role of TDP-43 in ALS and other TDP-43... 65K€ Cerrado

SAF2009-13015-C02-02 CRIBADO FARMACOLOGICO DE FARMACOS NEUROGENICOS COMO TRATAMIE... 81K€ Cerrado

FJC2021-046632-I New therapeutic approaches for ALS and others TDP-43-pathies 65K€ Cerrado

IJC2019-039229-I Targeting autophagy in amyotrophic lateral sclerosis and fro... 93K€ Cerrado

SAF2008-03001 EXCITOTOXICIDAD, PLEGAMIENTO ANOMALO DE PROTEINAS Y MECANISM... 169K€ Cerrado

SAF2015-64948-C2-1-R FARMACOS INNOVADORES PARA ENFERMEDADES NEURODEGENERATIVAS (A... 182K€ Cerrado

Duración del proyecto: 26 meses Fecha Inicio: 2024-03-12
Fecha Fin: 2026-05-31

Líder del proyecto

STICHTING RADBOUD UNIVERSITEIT No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 203K€

Descripción del proyecto Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by motor neuron degeneration that leads to progressive muscle atrophy. Currently only a few FDA-approved drugs are available for ALS, which merely extend patients’ life by a few months. Thus, there is an urgent need for new and more effective disease-modifying treatments. Although ALS is characterized by selective motor neuron degeneration, extensive evidence indicates that other cell types contribute to both onset and progression of ALS. An unresolved question in the ALS field is whether skeletal muscle fibers are merely bystanders or rather active contributors to motor neuron degeneration in ALS, and if so, through which biological pathways. Previous and ongoing work by the host laboratory has demonstrated the contribution of skeletal muscle in FUS-ALS, (1-2% of ALS cases). As TDP-43 cytoplasmic inclusions are a universal feature in nearly all ALS patients, assessing whether mutant TDP-43 toxicity in muscle contributes to ALS may be relevant to the broad community of ALS patients. I hypothesize that TDP-43 toxicity in skeletal muscle contributes to motor neuron degeneration and ALS-related phenotypes through specific molecular programs. I will use genetic and viral approaches to modulate mutant TDP-43 levels specifically in skeletal muscle in a TDP-43-ALS mouse model, and assess the effects on ALS-related phenotypes. Moreover, I will use single-nucleus RNA sequencing, a cutting-edge technique that became recently available, to determine – for the first time – the biological function of TDP-43 in skeletal muscle in the context of ALS, and to potentially identify novel therapeutic targets. Indeed, if mutant TDP-43 expression in skeletal muscle would influence the age of disease onset and/or disease progression, this would imply that therapeutic strategies for ALS should not only target motor neurons but also skeletal muscles, potentially improving ALS treatment approaches.

Conecta tu I+D

Entra hoy

¿Olvidó su contraseña?

Financiación

Empresas

CTIs/Universidades

Proyectos

Investigadores