Frontotemporal dementia is a clinically, genetically and pathologically hetero-geneous neurodegenerative disorder, enclosing a wide range of different pathological entities, associated with the accumulation of proteins such as tau and TPD-43. Characterized by a high hereditability, mutations in three main genes, MAPT, GRN and C9orf72, can drive the neurodegenerative process. The connection between different genes and proteinopathies through specific mechanisms has shed light on the pathophysiology of the disease, leading to the identification of potential pharmacological targets. New experimental strategies are emerging, in both preclinical and clinical settings, which focus on small molecules rather than gene therapy. In this review, we provide an insight into the aberrant mechanisms leading to FTLD-related proteinopathies and discuss recent therapies with the potential to ameliorate neurodegeneration and disease progression.

Experimental Disease-Modifying Agents for Frontotemporal Lobar Degeneration / M. Giunta, E. Solje, F. Gardoni, B. Borroni, A. Benussi. - In: JOURNAL OF EXPERIMENTAL PHARMACOLOGY. - ISSN 1179-1454. - 13:(2021 Mar 24), pp. 359-376. [10.2147/JEP.S262352]

Experimental Disease-Modifying Agents for Frontotemporal Lobar Degeneration

F. Gardoni;
2021

Abstract

Frontotemporal dementia is a clinically, genetically and pathologically hetero-geneous neurodegenerative disorder, enclosing a wide range of different pathological entities, associated with the accumulation of proteins such as tau and TPD-43. Characterized by a high hereditability, mutations in three main genes, MAPT, GRN and C9orf72, can drive the neurodegenerative process. The connection between different genes and proteinopathies through specific mechanisms has shed light on the pathophysiology of the disease, leading to the identification of potential pharmacological targets. New experimental strategies are emerging, in both preclinical and clinical settings, which focus on small molecules rather than gene therapy. In this review, we provide an insight into the aberrant mechanisms leading to FTLD-related proteinopathies and discuss recent therapies with the potential to ameliorate neurodegeneration and disease progression.
C9orf72; GRN; MAPT; TDP-43; frontotemporal dementia; frontotemporal lobar degeneration; tau; therapy
Settore BIO/14 - Farmacologia
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/829233
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