Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis.
Facioscapulohumeral muscular dystrophy (FSHD) is a muscle wasting disease caused by aberrant expression of double homeobox protein 4 (DUX4). When DUX4 is activated in skeletal muscle, it triggers myocyte cell death after several transcriptional changes, thus genetic DUX4 silencing arises as a promising approach for treating FHSD.
Korea Research Institute of Chemical Technology has prepared and tested new serine/threonine-protein kinase LATS1 and LATS2 inhibitors reported to be useful for the treatment of sarcopenia, muscular dystrophy, inflammatory myopathy, congenital myopathy, myotonia, myofascial pain syndrome, motor neuron diseases and muscle injury.
In the inflamed joints of rheumatoid arthritis, CD4+ T lymphocytes accumulate lipid droplets that make them vulnerable and promote their death, thereby amplifying joint inflammation. A study led by scientists at Mayo Clinic and Stanford University suggests that blocking the formation of these lipid droplets or their contents could offer a therapeutic strategy for this condition.
The variety of organoids that can be developed in vitro is enabling major advances. Depending on the type of tissues and the research goals, these small 3D cell-based structures that mimic real tissue offer certain advantages over animal models. Scientists at the University of Padova in Italy have created human neuromuscular organoids to reproduce cancer-induced muscle cachexia, a condition that murine models do not accurately replicate.
Researchers from the University of São Paulo (Brazil) first proposed using eccentric training as a promising intervention to address musculoskeletal impairments associated with Marfan syndrome. Eccentric training is a form of resistance exercise that focuses on muscle lengthening under load and can induce robust skeletal muscle adaptations, including the attenuation of muscle wasting, promotion of myofiber hypertrophy and stimulation of satellite cell activation and proliferation, as previously demonstrated.
Haisco Pharmaceutical Group Co. Ltd. has identified interleukin-17A (IL-17A) production inhibitors with reduced toxic and side effects reported to be useful for the treatment of arthritis, multiple sclerosis and psoriasis.
Alebund Pharmaceuticals (Hong Kong) Ltd. has described complement factor B (CFB) inhibitors reported to be useful for the treatment of glomerular disorders and rheumatoid arthritis.
Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, which is then converted by ENPP1 into AMP and pyrophosphate (PPi). Because PPi is a potent inhibitor of calcification, reduced systemic PPi production is a key driver of PXE. University of Pennsylvania investigators and collaborators proposed applying liver-targeted variant correction via genome editing as a single-intervention therapeutic approach for PXE, leading to subsequent restoration of systemic PPi.
Researchers from Stanford University have reported that inhibiting the enzyme 15-hydroxyprostaglandin dehydrogenase (15-PGDH) promoted cartilage regeneration in mouse models of osteoarthritis due to either aging or tissue injury. An oral version of the inhibitor that the team used is in a clinical trial for sarcopenia; it improved muscle mass and strength in preclinical studies. However, the mechanism by which 15-PDGH inhibition works appears to differ in the two conditions.
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