Young boys with Duchenne Muscular Dystrophy (DMD) experience poor muscle regeneration; however, the exact reasons for this remain under investigation. An experimental model of extreme DMD that suffers a big raise in reworking growth factor-beta (TGFβ) activity after muscle injury reveals that high TGFβ activity suppresses muscle regeneration and promotes fibroadipogenic progenitors (FAPs).
This results in the replacement of the broken muscle fibers by calcified and connective tissue, compromising muscle structure and performance. While blocking FAP buildup offers a partial answer, a Children’s National Hospital research crew identifies correcting the muscle micro-environment caused by high TGFβ as a ripe therapeutic target.
The group’s examine featured online March 26, 2020, in JCI Perception.
DMD is a chronic muscle illness that affects 1 in 6,200 younger males in the prime of their lives. The dysfunction, attributable to genetic mutations resulting in the inability to produce dystrophin protein, leads to ongoing muscle injury, chronic irritation and poor recovery of lost muscle tissue. The sufferers experience progressive muscle wasting, lose the ability to walk by the time they’re teenagers, and die prematurely resulting from cardiorespiratory failure.
The Children’s National group finds for the first time that as early as preadolescence, their experimental model of extreme DMD illness confirmed clear indicators of the type of spontaneous muscle injury, regenerative failure and muscle fiber loss witnessed in preadolescent boys who’ve DMD.