Michael
Gait

Therapeutic applications of synthetic oligonucleotide analogues & their peptide conjugates

Our work focuses on the chemical synthesis of modified oligonucleotides, siRNA, peptide nucleic acids (PNA), and their peptide conjugates to target essential RNA structures inside cells for a variety of potential therapeutic applications.

The following studies are currently in progress:

a) Steric block antisense targeting by PNA conjugated to cell penetrating peptides to mediate alternative RNA splicing in the cell nucleus. One application is exon skipping of the dystrophin gene in a mouse model of Duchenne Muscular Dystrophy, a genetic disease manifest in young boys for which there is currently no cure.

b) Synthesis of PNA and other analogues as steric block agents targeted to microRNA, important newly discovered regulators of gene expression, with applications for example in liver disease, cancer and neuromuscular diseases.