Advancing Precision RNAi for Fibrodysplasia Ossificans Progressiva Through a Combinatorial, Unimolecular, Allele-Selective Approach

• Combining a SNP-specific siRNA with an inflammatory target to tackle both the underlying genetic driver and disease progression in fibrodysplasia ossificans progressiva
• Developing multimeric oligonucleotide architectures to simultaneously address multiple disease-driving pathways
• Demonstrating therapeutic potential through both preventative and therapeutic models, including local and systemic delivery

Speakers:

Julia Alterman

Assistant professor

University of Massachusetts Medical School