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SOLID

Financiado

Suppression of Organelle Defects in Human Disease

The goal of SOLID is the systematic mapping of genetic suppression for important organelles with impaired fidelity in human disease to uncover new strategies to correct the perturbed state. Functioning of the eukaryotic cell relie... ver más

30/04/2025

LMU MUENCHEN

1M€

Presupuesto del proyecto: 1M€

Líder del proyecto

LUDWIGMAXIMILIANSUNIVERSITAET MUENCHEN No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Ver 2 Participantes

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2018-11-05

Línea de financiación objetivo El proyecto se financió a través de la siguiente ayuda:

ERC-2018-STG: ERC Starting Grant

I+D

Cerrada hace 7 años

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2 Participantes

LMU MUENCHEN

1.50M€ | Lider

NOVATTIA DESARROLLOS

224.62K€ | Participante

Últimas noticias

30-11-2024: Cataluña Gestión For... Se ha cerrado la línea de ayuda pública: Gestión Forestal Sostenible para Inversiones Forestales Productivas para el organismo:

29-11-2024: IDAE En las últimas 48 horas el Organismo IDAE ha otorgado 4 concesiones

29-11-2024: ECE En las últimas 48 horas el Organismo ECE ha otorgado 2 concesiones

Duración del proyecto: 77 meses Fecha Inicio: 2018-11-05
Fecha Fin: 2025-04-30

Líder del proyecto

LUDWIGMAXIMILIANSUNIVERSITAET MUENCHEN

TRL 4-5

Presupuesto del proyecto 1M€

Descripción del proyecto The goal of SOLID is the systematic mapping of genetic suppression for important organelles with impaired fidelity in human disease to uncover new strategies to correct the perturbed state. Functioning of the eukaryotic cell relies on the concerted activity of biological machines, defects in which can cause incurable human disease, including metabolic disorders and neurodegeneration. This is exemplified by malfunctioning mitochondria or lysosomes, for which (alongside environmental triggers) hundreds of causative genes have been identified, but cures are mostly lacking. This is mainly due to the difficulty of drugging loss-of-function mutations, which uniquely alter or entirely remove the gene product. Extensive studies in yeast and initial work in the human system show that cellular modules contain a substantial amount of hidden genetic suppression. Revealing suppressors of disease-defining features of organelle perturbation and targeting those instead of the query mutations, will address the frustrations of drugging an absent factor and etiological diversity. Until now, mutation-based genome-wide suppressor surveys were mostly limited to model organisms and phenotypes yielding growth defects, due to technical hurdles associated with recessive genetics in the human system. However, with the development of a groundbreaking new forward genetics platform that combines genome engineering, ultradeep haploid mutagenesis and direct stratification of fixed mutants, we can now, for the first time, interrogate genetic suppression of human organelle defects independent of cellular fitness. SOLID will also develop new technology to study organelle fidelity in an exhaustive fashion that departs from a gene-centric view point and captures complex phenotypes. Altogether, this will create a new paradigm of 'studying cell biology via genetics' widely applicable to many different biological questions, and reveal urgently needed novel therapeutic possibilities for incurable diseases.

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