Hong Moulton

Professor, Biological Chemistry/Antisense Therapy


Morpholino oligomers are a class of steric-blocking antisense molecules that have been widely used to knock down gene expression, modify pre-mRNA splicing or inhibit miRNA maturation and activity. Injection of Morpholinos into single-celled embryos of many creatures results in specific knockdown of targeted genes with little toxicity. Morpholino oligomers have revolutionary potential for treatment of a broad range of human diseases, including viral, bacterial, age-related and genetic diseases, but they suffer from poor delivery into cells. My long term research interest has been in inventing and improving methods for enhancing in vivo delivery of Morpholinos in a tissue-specific manner for a giving disease by chemically modifying and/or formulating Morpholinos. My current research is 1) to develop and validate a high throughput in vivo model to assess the efficacy and toxicity of intracellular delivery methods for steric-blocking antisense oligos, and 2) to investigate how host factors affect influenza viral infection using the Morpholino-mediated gene knockdown approach.