Niki Sepanj

Allosteric Modulation of Pathological Ataxin‐3 Aggregation: A Path to Spinocerebellar Ataxia Type‐3 Therapies (Adv. Sci. 11/2026)

Alexandra Silva, Sara Duarte‐Silva, Pedro Miguel Martins, Beatriz Rodrigues et autres

Allosteric Modulation of Pathological Ataxin‐3 Aggregation: A Path to Spinocerebellar Ataxia Type‐3 Therapies

Alexandra Silva, Sara Duarte‐Silva, Pedro Miguel Martins, Beatriz Rocha Ilidio Rodrigues et autres

Spinocerebellar ataxia type 3 (SCA3) is a rare neurodegenerative disorder caused by the expansion of a polyglutamine (polyQ) repeat in ataxin-3 (Atx3) for which no disease-modifying therapies are available. The presence of protein inclusions enriched in polyQ-expanded Atx3 in neurons suggests that …

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Allosteric Modulation of Pathological Ataxin-3 Aggregation: A Path to Spinocerebellar Ataxia Type-3 Therapies

Alexandra Silva, Sara Duarte‐Silva, Pedro Miguel Martins, Beatriz Rodrigues et autres

Spinocerebellar ataxia type 3 (SCA3) is a rare inherited neurodegenerative disease caused by the expansion of a polyglutamine repeat in the protease ataxin-3 (Atx3). Despite extensive knowledge of the downstream pathophysiology, no disease-modifying therapies are currently available to halt disease progression. The …

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Examination of SOD1 aggregation modulators and their effect on SOD1 enzymatic activity as a proxy for potential toxicity

Ravinder Kumar Malik, Christian I. Corrales, Miriam Linsenmeier, Huda Alalami et autres

Small-molecule inhibitors of abnormal protein self-assembly are promising candidates for developing therapy against proteinopathies. Such compounds have been examined primarily as inhibitors of amyloid β-protein (Aβ), whereas testing of inhibitors of other amyloidogenic proteins has lagged behind. An important issue with screening …

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The molecular tweezer CLR01 inhibits aberrant superoxide dismutase 1 (SOD1) self-assembly in vitro and in the G93A-SOD1 mouse model of ALS

Ravinder Kumar Malik, Helen M. L. Meng, Piriya Wongkongkathep, Christian I. Corrales et autres

Mutations in superoxide dismutase 1 (SOD1) cause 15–20% of familial amyotrophic lateral sclerosis (fALS) cases. The resulting amino acid substitutions destabilize SOD1's protein structure, leading to its self-assembly into neurotoxic oligomers and aggregates, a process hypothesized to cause the characteristic motor-neuron degeneration …

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