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.
ABSTRACT
Drugs for the treatment of rheumatoid arthritis ( RA) include disease-modifying antirheumatic drugs ( DMARDs ), nonsteroidal anti-inflammatory drugs ( NSAIDs) and biological agents, etc. These drugs are critical in preventing the process and complications of RA. However, the outcome of treatment and adverse drug reactions with these drugs in RA patients are different individually. Drug-metabolizing enzymes (dihydrofolate reductase, cytochrome P450enzymes, N-acetyltransferases, and so on. ), drug transporters ( ATP-binding cassette transporters) and drug targets ( tumor necrosis factor-α receptors) are coded for by variant alleles. The gene polymorphism of drug transport-ers can change the distribution and excretion of drugs. The poly-morphisms of drug target affect significantly drug sensitivity. These gene polymorphisms may influence the pharmacokinetics, pharmacodynamics and side effects of medicine. In this article, we review the genetic polymorphisms that affect the efficacy of drug or the occurrence of adverse drug reactions in RA.