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Developing an ON/OFF switch for gene therapy

Written by Dr Hannah K Shorrock Edited by Dr Larissa Nitschke Splice modifying compounds can regulate whether or not gene therapy is active Gene therapy is an incredibly versatile therapeutic strategy that can be used to treat monogenic disorders. Gene therapy can inactivate or silence mutant gene transcripts, edit the Read More…

Developing therapeutically relevant biomarkers for SCA3

Written by Dr Hannah K Shorrock Edited by Dr Vitaliy V Bondar Measuring levels of polyQ ATXN3 proteins in cerebrospinal fluid and plasma distinguishes SCA3 patients from unaffected individuals For disease-modifying medicines or therapies to be approved for use in the clinic, the treatments must demonstrate success at meeting pre-set Read More…

Dieta rica em Creatina atrasa a SCA3 em camundongos

Escrito por Dr. Lauren R. Moore Editado por Dr.Larissa Nitschke. Inicialmente publicado em 5 de julho de 2019. Traduzido para o português por Ana Carolina Martins. A creatina, um suplemento alimentar comum tomado por atletas, atrasa sintomas e melhora o equilíbrio e força em camundongos com SCA3. Poderia um suplemento Read More…

Identifying serotonin receptors as a therapeutic target for SCA3

Written by Dr. Hannah K Shorrock Edited by Dr. Hayley McLoughlin A C. elegans model of SCA3 helps to identify the serotonin receptor agonist befiradol as a potential therapeutic for SCA3 To move toward treatments for ataxia patients, it is important to understand what cellular and molecular pathways are dysfunctional Read More…

Measuring neurodegeneration in spinocerebellar ataxias

Written by Dr Hannah K Shorrock Edited by Dr. Maria do Carmo Costa Neurofilament light chain predicts cerebellar atrophy across multiple types of spinocerebellar ataxia A team led by Alexandra Durr at the Paris Brain Institute identified that the levels of neurofilament light chain (NfL) protein are higher in SCA1, Read More…

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