Amyotrophic Lateral Sclerosis (ALS): Pathogenesis and Treatments
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Neurobiology".
Deadline for manuscript submissions: 20 October 2025 | Viewed by 2720
Special Issue Editors
Interests: amyotrophic lateral sclerosis; motor neuron disease
2. Brain-Targeted Nanotechnologies (BraiNs) Lab, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
Interests: microRNA; cell biology; neurobiology; neurodegenerative diseases
Special Issue Information
Dear Colleagues,
As you know, amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease marked by significant clinical heterogeneity. This heterogeneity is reflected in variations in patient age at onset, the type of motor neuron involvement, patterns of symptom onset and progression, and the presence of comorbidities, including cognitive and behavioral changes, autonomic dysfunction, and sensory involvement.
Despite extensive research, the pathogenesis of ALS remains unclear. However, studies have shown the involvement of multiple altered signaling pathways, including mitochondrial dysfunction, glutamate excitotoxicity, oxidative stress, and neuroinflammation. For understanding this heterogeneity and for the development of new therapeutic approaches, the identification of specific molecular mechanisms leading to motoneurons degeneration and extra-motorneuron involvement is crucial in ALS. Consistently, the contributions of RNA metabolism, protein misfolding, and neuroinflammation require further study, particularly taking into consideration disease heterogeneity.
A deeper understanding of ALS heterogeneity across multiple levels is essential for tailoring treatments more precisely. Currently, few targeted therapies address specific ALS subtypes, and identifying which patients benefit from particular interventions remains a challenge. The potential of deep phenotyping, which integrates detailed clinical data with molecular and genetic insights, holds great promise. This approach could help uncover biomarkers for early diagnosis, monitor treatment responses, and ultimately pave the way for more personalized and effective therapies.
This Special Issue aims to highlight the key pathological mechanisms underlying the diverse ALS phenotypes and explore emerging therapeutic strategies targeting these pathways.
Dr. Monica Consonni
Dr. Stefania Marcuzzo
Guest Editors
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Keywords
- amyotrophic lateral sclerosis
- therapeutic applications
- extracellular-vesicles
- gene variants
- RNA metabolism
- mitochondrial dysfunction
- oxidative stress
- neuroinflammation
- protein misfolding
- muscle impariment
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