Antisense drug research and development
Antisense drug delivery
Host and pathogen interactions
Neuromuscular and infectious diseases
Aquatic models for human and animal diseases
I work on improving delivery of Morpholino oligos into cells. 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 across the cell membrane 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 neuromuscular and infectious diseases. I am characterizing an engineered zebrafish designed as an antisense oligo activity reporter system and plan to use this fish to test oligo delivery moieties and formulations.
I am interested in working in collaborative projects to synthesize delivery-enhanced Morpholino oligos for treatment of disease models in organisms. I am currently funded by a NIH-funded research project studying influenza virus host factors and am actively pursuing funding to develop oligo-peptide conjugates to inhibit or eliminate infection with Toxoplasma.
Selected Publications: http://www.ncbi.nlm.nih.gov/pubmed/?term=moulton+h