Article

Genotype–Phenotype Distinctions in Spastic Paraplegia 4 Reveal HDAC6 as a Therapeutic Target

2025-07-18

Abstract excerpt

Spastic Paraplegia 4 (SPG4) is the most prevalent form of Hereditary Spastic Paraplegia (HSP), a neurodegenerative disorder characterized by progressive lower limb spasticity and debilitating gait impairment, primarily driven by axonal degeneration of corticospinal motor neurons (CSMNs). Caused by mutations in the SPAST gene encoding spastin, an AAA-ATPase involved in microtubule severing and intracellular organe...

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Literature Corpus work: d78f9039-d91f-5f8f-9374-a86b6405c64f
DOI: 10.1101/2025.07.15.664947

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