Advance in animal model and therapeutic drugs for progressive muscular dystrophy / 中国药理学通报

ABSTRACT

Progressive muscular dystrophy is an X-linked recessive hereditary degenerative disease caused by dystrophin gene defects, and there is currently no effective treatment.With the further study of progressive muscular dystrophy, a series of animal models have been developed to evaluate the efficacy of drugs, such as muscular dystrophy protein deficiency mice, double gene knockout Duchenne muscular dystrophy phenotype mice, muscular dystrophy dogs and zebrafish muscular dystrophy models.A variety of therapeutic strategies and dmgs are under development, such as inhibition of nonsense mutations, exon hopping therapy, gene therapy, calcium toxicity relieving drugs and antioxidants.This article reviews the pathogenesis, establishment and evaluation of animal models and the therapeutic drngs of progressive muscular dystrophy.