LGALS3 identified as Huntington disease target

INDICATION: Huntington disease (HD)

Inhibiting the lectin LGALS3 could treat HD, which is caused by aggregation of mutant HTT. Plasma levels of LGALS3 protein were higher in 26 symptomatic HD patients than in four presymptomatic and 16 non-HD individuals. Moreover, high plasma LGALS3 levels were associated with increased disease burden and reduced cognitive and neurological function among the symptomatic patients. In postmortem samples, LGALS3 mRNA levels were higher in the caudate and putamen of five HD patients than in five healthy individuals. In a mouse model of HD, a lentiviral vector encoding shRNA against LGALS3 improved motor performance, extended lifespan and decreased the number of mutant HTT aggregates in the striatum. Next steps could include developing LGALS3 inhibitors that can cross the blood-brain barrier...