Baxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA 94305, USA.
Center for Genetic Disorders and Aging, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.
Sci Transl Med. 2023 Oct 11;15(717):eadg1485. doi: 10.1126/scitranslmed.adg1485.
To date, there are no approved treatments for the diminished strength and paralysis that result from the loss of peripheral nerve function due to trauma, heritable neuromuscular diseases, or aging. Here, we showed that denervation resulting from transection of the sciatic nerve triggered a marked increase in the prostaglandin-degrading enzyme 15-hydroxyprostaglandin dehydrogenase (15-PGDH) in skeletal muscle in mice, providing evidence that injury drives early expression of this aging-associated enzyme or gerozyme. Treating mice with a small-molecule inhibitor of 15-PGDH promoted regeneration of motor axons and formation of neuromuscular synapses leading to an acceleration in recovery of force after an acute nerve crush injury. In aged mice with chronic denervation of muscles, treatment with the 15-PGDH inhibitor increased motor neuron viability and restored neuromuscular junctions and function. These presynaptic changes synergized with previously reported muscle tissue remodeling to result in a marked increase in the strength of aged muscles. We further found that 15-PGDH aggregates defined the target fibers that are histopathologic hallmarks of human neurogenic myopathies, suggesting that the gerozyme may be involved in their etiology. Our data suggest that inhibition of 15-PGDH may constitute a therapeutic strategy to physiologically boost prostaglandin E2, restore neuromuscular connectivity, and promote recovery of strength after acute or chronic denervation due to injury, disease, or aging.
迄今为止,对于因创伤、遗传性神经肌肉疾病或衰老导致的周围神经功能丧失而导致的力量减弱和瘫痪,还没有批准的治疗方法。在这里,我们表明,坐骨神经横断导致的去神经作用会在小鼠的骨骼肌中引发前列腺素降解酶 15-羟基前列腺素脱氢酶(15-PGDH)的显著增加,这为损伤驱动这种与衰老相关的酶或“衰老酶”的早期表达提供了证据。用 15-PGDH 的小分子抑制剂治疗小鼠,可促进运动轴突的再生和形成神经肌肉突触,从而加速急性神经挤压损伤后的力量恢复。在慢性肌肉去神经的老年小鼠中,用 15-PGDH 抑制剂治疗可增加运动神经元的存活率,并恢复神经肌肉接头和功能。这些突触前变化与之前报道的肌肉组织重塑协同作用,导致老年肌肉力量显著增加。我们进一步发现,15-PGDH 聚集体定义了目标纤维,这些纤维是人类神经源性肌病的组织病理学特征,表明该衰老酶可能与其病因有关。我们的数据表明,抑制 15-PGDH 可能构成一种治疗策略,可在生理上增强前列腺素 E2,恢复神经肌肉连接,并促进因损伤、疾病或衰老导致的急性或慢性去神经后的力量恢复。