Simon C. Warby

In situ targeted base editing of bacteria in the mouse gut

Antisense oligonucleotide therapeutic approach for Timothy syndrome

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup