Novel probes of BMP and TGF-beta signaling and function

NIH RePORTER · NIH · R01 · $266,477 · view on reporter.nih.gov ↗

Abstract

PROJECT SUMMARY/ABSTRACT Heterotopic ossification (HO), the formation of ectopic endochondral bone in skeletal muscle and soft tissues, is a significant cause of morbidity from joint immobility and pain. The precise mechanisms responsible for HO remain incompletely understood; however, the association with genetic gain-of- function syndromes affecting receptors and ligands of the bone morphogenetic protein (BMP) and activin signaling pathways has generated important insights and translatable strategies. We have previously identified activin A as the dominant ligand responsible for the congenital HO syndrome fibrodysplasia ossificans progressiva (FOP), being transduced via mutant ACVR1 encoding ALK2. In subsequent work using a ligand trap, we and collaborators found that ligands of the BMP type I receptor ALK3 account for a major portion of non-genetic forms of HO, frequently complicating joint replacement surgeries, burn injuries, blast trauma, or inflammatory arthropathies. In the parent grant for this supplement we describe several pharmacologic and genetic strategies for narrowing the identify of ligands and receptors responsible for non-genetic forms of HO, using genetically modified human derived tissue cultures and genetically modified mouse reagents and tissues. Here we propose to generate a panel of novel reagents that can selectively target signaling via specific type I and type II receptors in this pathway in non-transfected or non-engineered tissues, and thus permit complementary studies in unmodified human and rodent tissues without artifacts due to genetic modification or off-target effects confounding interpretation. Moreover, these antibody-based reagents would have pharmacokinetics suitable for in vivo validation studies and could be rapidly translated into therapies for tissue regeneration or orthopedic applications. Importantly. these studies will provide a robust set of tools that can be used to further elucidate the molecular blueprint for development, wound healing, as well as HO involving skeletal muscle and tendon niches, and create a broad platform for scientific discovery and translational work in musculoskeletal disease.

Key facts

NIH application ID
10051129
Project number
3R01AR057374-07S1
Recipient
BRIGHAM AND WOMEN'S HOSPITAL
Principal Investigator
PAUL B YU
Activity code
R01
Funding institute
NIH
Fiscal year
2020
Award amount
$266,477
Award type
3
Project period
2019-07-01 → 2021-06-30