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Collagen type VI regulates TGFβ bioavailability in skeletal muscle

Mohassel, P.; Rooney, J.; Zou, Y.; Johnson, K.; Norato, G.; Hearn, H.; Nalls, M. A.; Yun, P.; Ogata, T.; Silverstein, S.; Sleboda, D.; Roberts, T. J.; Rifkin, D. B.; Bonnemann, C. G.

2023-06-24 neuroscience
10.1101/2023.06.22.545964 bioRxiv
Show abstract

Collagen VI-related disorders (COL6-RDs) are a group of rare muscular dystrophies caused by pathogenic variants in collagen VI genes (COL6A1, COL6A2, and COL6A3). Collagen type VI is a heterotrimeric, microfibrillar component of the muscle extracellular matrix (ECM), predominantly secreted by resident fibroadipogenic precursor cells in skeletal muscle. The absence or mislocalizatoion of collagen VI in the ECM underlies the non-cell autonomous dysfunction and dystrophic changes in skeletal muscle with an as of yet elusive direct mechanistic link between the ECM and myofiber dysfunction. Here, we conduct a comprehensive natural history and outcome study in a novel mouse model of COL6-RDs (Col6a2-/-mice) using standardized (Treat-NMD) functional, histological, and physiologic parameter. Notably, we identify a conspicuous dysregulation of the TGF{beta} pathway early in the disease process and propose that the collagen VI deficient matrix is not capable of regulating the dynamic TGF{beta} bioavailability at baseline and also in response to muscle injury. Thus, we propose a new mechanism for pathogenesis of the disease that links the ECM regulation of TGF{beta} with downstream skeletal muscle abnormalities, paving the way for developing and validating therapeutics that target this pathway.

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