Haiyan Zhou

This study evaluates cell-specific activity of antisense oligonucleotide conjugates in vivo using a reporter mouse model and finds that palmitate conjugation shows broad extrahepatic activity and preferential skeletal muscle targeting.

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.

The paper identifies early translational defects in SMA mice, showing reduced translational efficiency and ribosome numbers in motor neurons, which are rescued by SMN restoration.