From basic research to the clinic: innovative therapies for ALS and FTD in the pipeline
Autor: | Maurizio Romano, Rajka M. Liscic, Nigel J. Cairns, Emanuele Buratti, Antonella Alberici |
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Přispěvatelé: | Liscic, R. M., Alberici, A., Cairns, N. J., Romano, M., Buratti, E. |
Rok vydání: | 2019 |
Předmět: |
0301 basic medicine
medicine.medical_specialty Neurology Dementia FTLD Review lcsh:Geriatrics lcsh:RC346-429 03 medical and health sciences Cellular and Molecular Neuroscience 0302 clinical medicine Genetic Basic research medicine Genetics Effective treatment Humans Amyotrophic lateral sclerosis Molecular Biology lcsh:Neurology. Diseases of the nervous system Motor neuron disease TDP-43 Stem cell business.industry Therapies Investigational Amyotrophic Lateral Sclerosis Cognition Neurodegenerative Diseases FTD medicine.disease Molecular medicine lcsh:RC952-954.6 030104 developmental biology Innovative Therapies Frontotemporal Dementia Mutation Neurology (clinical) Personalized medicine ALS Atrophy business Neuroscience 030217 neurology & neurosurgery |
Zdroj: | Molecular Neurodegeneration Molecular Neurodegeneration, Vol 15, Iss 1, Pp 1-17 (2020) |
ISSN: | 1750-1326 |
Popis: | Amyotrophic lateral sclerosis (ALS) and Frontotemporal Degeneration (FTD) are neurodegenerative disorders, related by deterioration of motor and cognitive functions and short survival. Aside from cases with an inherited pathogenic mutation, the causes of the disorders are still largely unknown and no effective treatment currently exists. It has been shown that FTD may coexist with ALS and this overlap occurs at clinical, genetic, and molecular levels. In this work, we review the main pathological aspects of these complex diseases and discuss how the integration of the novel pathogenic molecular insights and the analysis of molecular interaction networks among all the genetic players represents a critical step to shed light on discovering novel therapeutic strategies and possibly tailoring personalized medicine approaches to specific ALS and FTD patients. |
Databáze: | OpenAIRE |
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