From Genetic Risk to Targeted Treatment in Neurodegenerative Disorders

Neurodegenerative disorders, including Alzheimer's disease, Parkinson's disease, and Amyotrophic Lateral Sclerosis, constitute a primary global health concern due to their progressive, debilitating, and currently incurable nature. These diseases arise from extensive neuronal degeneration, impairing motor, cognitive, and autonomic functions. Advances in molecular biology and genomics have catalyzed a paradigm shift from symptomatic treatment to mechanistic understanding, emphasizing the etiological role of genetic factors. Central to this shift is the emerging utility of genetic signatures—distinctive gene expression profiles that reflect underlying pathogenic processes. These signatures include coordinated transcriptional changes across gene networks, offering enhanced resolution over single-gene analyses. In the context of neurodegeneration, they hold significant potential for early detection, prognostication, and the development of targeted, pre-symptomatic interventions. The strategic research objective is to establish predictive biomarkers capable of identifying at-risk individuals before clinical onset, thereby facilitating the development of preventive or disease-modifying therapies.Genetic signatures can be classified into prognostic, diagnostic, and predictive categories. This classification outlines a precise, strategic framework for implementing precision medicine in neurodegenerative disorders. Prognostic signatures identify who is at risk or what the likely disease course will be; diagnostic signatures clarify what specific disease or subtype a patient has; and crucially, predictive signatures determine how a patient is expected to respond to which particular treatment. This systematic classification allows clinicians to move beyond a "one-size-fits-all" approach to tailored therapies, optimizing treatment selection, improving patient outcomes, and potentially streamlining healthcare resource allocation by avoiding ineffective treatments. The discovery and characterization of genetic signatures in neurodegenerative disorders are critically dependent on the continuous advancement and synergistic application of high-throughput genomic technologies, large-scale genetic studies, and sophisticated computational and bioinformatics approaches. By embracing this integrated, multidisciplinary approach, the field is charting a course towards a future where neurodegenerative disorders are not only better understood but also effectively prevented, precisely diagnosed, and successfully treated, ultimately enhancing the quality of life for millions worldwide.The current Research Topic aims to cover promising, recent, and novel research trends in the knowledge of genetic signatures in neurodegenerative disorders. Areas to be covered in this Research Topic may include, but are not limited to:- Multidisciplinary investigation of patient cohorts - Evaluation of molecular pathways of disease - Precision medicine for neurodegenerative disorders- Development or assessment of treatment strategies - Investigation of new biomarkers (prognostic, diagnostic, and predictive) Topic editors: Carlo Caltagirone, Stefania Zampatti, Benedetta Nacmias Source: https://www.frontiersin.org/research-topics/71918/from-genetic-risk-to-targeted-treatment-in-neurodegenerative-disorders