December 2, 2024 at 12:30 pm US EST
Fibrodysplasia Ossificans Progressiva (FOP) is a devastating, ultra-rare condition, where patients acquire bone in muscle and connective tissue either spontaneously or in response to injury. Caused by a mutation in the ACVR1 receptor, and exacerbated by inflammatory response, excessive bone grown means patients with FOP do not often live past the age of 40. Using an siRNA chemical architecture ideal for delivery to muscle, we have developed a combinatorial treatment for FOP targeting both the mutant ACVR1 receptor and an inflammatory cytokine. This approach supports significant reduction in bone growth in a mouse model of disease and may provide a promising new therapy for the treatment of FOP.
Dr. Julia Alterman is an Assistant Professor at the RNA Therapeutics Institute, UMass Chan Medical School, where she focuses on developing chemically durable, safe, and effective therapeutic oligonucleotides for extrahepatic delivery. Julia received her Ph.D. from UMass Med, and prior to joining the faculty, spent 7 years in biotech in the Boston area. While her PhD was in the field of oligonucleotide delivery to the CNS, she and her team are now working on targeting skin, muscle, bone, and eye, as well as designing and identifying hyper-functional sequences for specific genetic targets. Julia has published many peer-reviewed articles and holds several patents in the fields of oligonucleotide chemical biology and pharmacology.
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