A polytherapy approach demonstrates therapeutic efficacy for the treatment of SOD1 associated amyotrophic lateral sclerosisResearch in context

Summary: Background: SOD1 mutations are a significant contributor of familial amyotrophic lateral sclerosis (ALS) cases.SOD1 mutations increase the propensity for the protein to misfold and aggregate into insoluble proteinaceous deposits within motor neurons and neighbouring cells.The small molecule, CuATSM, has repeatedly shown in mouse models to

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The PPLD has advantages over conventional regression methods in application to moderately sized genome-wide association studies.

In earlier work, we have developed and evaluated an alternative approach to the analysis of SPEAR GWAS data, based on a statistic called the PPLD.More recently, motivated by a GWAS for genetic modifiers of the X-linked Mendelian disorder Duchenne Muscular Dystrophy (DMD), we adapted the PPLD for application to time-to-event (TE) phenotypes.Because

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