Sara Aguti

The paper identifies fibroblast-homing peptides targeting PDGFR for targeted delivery of nucleic acid therapeutics, showing enhanced efficacy in a collagen VI-related muscular dystrophy model.

The paper presents a framework for prioritizing targets and patients for antisense oligonucleotide therapies in rare diseases, focusing on equitable and systematic decision-making.

This paper introduces dasper, an R/Bioconductor package that detects pathogenic splicing events from RNA-sequencing data with higher accuracy than existing methods.