1.Advancements in mechanisms and drug treatments for fibrodysplasia ossificans progressiva.
Yijun ZHOU ; Ce SHI ; Hongchen SUN
Journal of Zhejiang University. Science. B 2025;26(4):317-332
Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by congenital bilateral malformation of the large toe and progressive, extensive, and irreversible heterotopic ossification (HO) of soft tissues throughout the body, leading to severe disabilities. FOP is caused primarily by mutations in activin A receptor type 1 (ACVR1), also known as activin-like kinase 2 (ALK2), which encodes a receptor belonging to the bone morphogenetic protein (BMP) type I family. However, the continuous and complex process of HO in FOP is not yet fully understood, which has impeded the development of therapeutic drugs. Despite surgical removal of HO, which often results in recurrence and expansion of ossification, there is currently no definitive drug treatment available to completely prevent, halt, or reverse the progression of HO in FOP. Currently, researchers are intensively studying the pathogenesis of FOP at various stages and developing promising drug candidates, including saracatinib, palovarotene, and rapamycin. This review provides an overview of progress in understanding the mechanism of FOP and the development of therapeutic drugs, with the goal of providing insights for further research and the development of new treatment methods.
Myositis Ossificans/genetics*
;
Humans
;
Activin Receptors, Type I/genetics*
;
Ossification, Heterotopic
;
Mutation
;
Sirolimus/therapeutic use*
;
Quinolones/therapeutic use*
;
Benzodioxoles/therapeutic use*
;
Animals
;
Quinazolines/therapeutic use*
2.Studies of effective part group of piperine to regulating lipid.
Xue-Mei BAO ; Sheng-Sang NA ; Jing-Kun LU
China Journal of Chinese Materia Medica 2013;38(6):909-913
The effects of effective part group on hyperlipidemia in animal were studied. The SD rats, hamsters and Kunming mouse were divided into blank group, model group. The positive control group and test group were fed with normal diet, blank and other groups were fed with high fat diet (mouse only a single intraperitoneal injection of egg yolk ). The corresponding concentration of solvent, simvastatin, effective part group of emulsion were given gavage once daily. The animal serum total cholesterol (TC) , triglyceride (TG) , low density lipoprotein (LDL) , high density lipoprotein (HDL) and liver TC, TG contents were determined to observe the effects of the effective fractions on blood lipid regulating function. Comparing with control group, the animial hyperlipidemia models of the SD rat (TC increase), mouse (TC, TG, LDL increase), hamsters ( TC, TG, LDL increase, HDL decrease) (P <0. 05, P < 0. 001) were successfully established. Piper longum effective part group could decrease the serum TC, TG, LDL (P <0.05, P < 0. 001) and liver TC, TG content, and elevate serum HDL levels (P <0.05, P <0.001). The golden hamster is ideal for hyperlipidemia model.
Alkaloids
;
pharmacology
;
therapeutic use
;
Animals
;
Benzodioxoles
;
pharmacology
;
therapeutic use
;
Cholesterol
;
blood
;
metabolism
;
Cricetinae
;
Drugs, Chinese Herbal
;
pharmacology
;
therapeutic use
;
Female
;
Hyperlipidemias
;
blood
;
drug therapy
;
metabolism
;
Lipid Metabolism
;
drug effects
;
Lipoproteins, HDL
;
blood
;
metabolism
;
Lipoproteins, LDL
;
blood
;
metabolism
;
Liver
;
drug effects
;
metabolism
;
Male
;
Mice
;
Piper
;
chemistry
;
Piperidines
;
pharmacology
;
therapeutic use
;
Polyunsaturated Alkamides
;
pharmacology
;
therapeutic use
;
Rats
;
Triglycerides
;
blood
;
metabolism

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