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Research Center for ALS

The Research Center for Amyotrophic Lateral Sclerosis (ALSCenter) brings together the groups that are involved in ALS research at the Institute, from an epidemiological, preclinical and translational point of view. It also coordinates the regional population-based registry of ALS and carries out activities in support to clinical studies.
 
ALS is a severe neurodegenerative disease of adulthood that affects motor neurons, the cells responsible for muscle contraction, a condition that leads to paralysis and consequent atrophy of the voluntary muscles of the whole body, finally involving the respiratory muscles. A therapy for ALS to date has not yet been developed. Only in about 10% of patients the disease is due to genetic mutations, in most cases the cause is unknown. It is hypothesized that ALS is caused by a combination of factors: genetic predisposition, environmental factors and processes associated with aging. ALSCenter research aims to understand which of these elements are the most important to hit, how and when, in order to stop or slow down the disease. It also aims to identify biomarkers to support diagnosis and prognosis and useful for monitoring the progression of the disease and the efficacy of therapies.

Obiettivi

Research lines

  • Study of pathogenetic mechanisms in experimental models to identify new and more effective drug targets,
  • Role of neuroinflammation and immune response,
  • Study of muscle atrophy in in vitro and in vivo models;
  • Development and implementation of research methods and tools (animal and cellular models, biochemical assays, innovative technologies, trial designs);
  • Identification and validation of biomarkers in preclinical and clinical studies;
  • Epidemiological studies on risk and prognostics factors, quality of life and outcomes;
  • Identification of molecules to test in experimental clinical studies.

approcci metodologici

Services offered

  • Pharmacological studies in animal models;
  • In vivo assessment of muscle atrophyusing MyoRep technology;
  • Analysis of biomarkers in biofluids for observational and interventionalclinical studies;
  • Support activities for observational and interventional clinical studies(CRO);
  • Statistical analysis for clinical studies;
  • Biobanking of samples from patients and animal models.
Progetti in corso

GATTALS - A gene therapy approach targeting TDP-43 pathology in ALS

Ente Finanziatore: AriSLA- Fondazione Italiana di Ricerca per la SLA

 

EVTestInALS - Extracellular vesicles in ALS: testing their use as biomarkers for prognosis and disease progression

Ente Finanziatore: AriSLA- Fondazione Italiana di Ricerca per la SLA

 

A mechanism-based gene therapy approach forTDP-43 proteinopathies: a proof-of-concept study in frontotemporal dementia and traumatic brain injury

Ente Finanziatore: Fondazione Cariplo

TDP-43 pathology and Lys-acetylation in circulating lymphomonocytes as biomarkers and therapeutic hints for amyotrophiclateral sclerosis

Ente Finanziatore: Ministero della Salute, Ricerca Finalizzata

 

Understanding the role of peripheral monocyteson muscle regeneration coupled to Amyotrophic Lateral Sclerosis progression

Ente Finanziatore: Ministero della Salute, Ricerca Finalizzata- StartingGrant

 

Intramuscular allosteric agonism of purinergic P2X7 receptor as a pharmacological approach to enhance skeletal muscleregeneration in Amyotrophic Lateral Sclerosis; Drug Development Grants

Ente Finanziatore: FightMND Australia

 

Dissecting the role of the myokine musclin against amyotrophic lateral sclerosis-induced muscle atrophy

Ente finaziatore: AFM Association Française Contre Les Myopathies -Trampoline Grant

 

What ALS wants?/Cosa vuole la SLA? A prospectivecohort study investigating the satisfaction of needs and priorities of caregivers and patients in relation to the administered care

Ente Finanziatore: Ministero della Salute, Ricerca Finalizzata Giovaniricercatori

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