Matthew R. Hassler

Divalent siRNA 2439-s4 lowers human PrP to 17% residual in transgenic mouse brain after one dose, and a separate mouse Prnp-targeting siRNA extends survival in prion-infected mice.

The paper shows that silencing SOD1 with optimized di-siRNA extends survival and prevents disease progression in ALS mice.

This study evaluates tetravalent siRNA configurations for photoreceptor gene silencing, finding that tetravalent siRNA provides robust, long-lasting efficacy with a good safety profile in rodent and swine models.

This paper demonstrates allele-specific siRNA silencing of mutant huntingtin in CNS using SNP-based targeting in vitro and in vivo.

This study shows that silencing ApoE in the brain with divalent siRNAs reduces amyloid plaques in Alzheimer's mice without affecting systemic cholesterol.