Advancements in gene therapy for Duchenne muscular dystrophy
Duchenne muscular dystrophy(DMD)is an X-linked recessive genetic disease caused by mutations in the DMD gene.This gene is the largest known gene in humans,consisting of 79 exons that encode a dystrophin protein containing 3685 amino acids.The size of the DMD gene contributes to the complexity and heterogeneity of mutation types,presenting challenges for the development of therapeutic drugs.Significant progress has been made in recent years toward the treatment of this disease,with several novel drugs approved for clinical development in various regions of the world as research advances.However,there is currently no cure for this disease.This review summarizes the new research progress of DMD gene therapy,and focuses on intro-ducing the most widely available codon reading and exon skipping therapies and their therapeutic mechanisms.In addition,the issues of clinical endpoint selection and accessibility of drug information in DMD new drug research were discussed,as well as the challenges and challenges currently faced by gene therapy,providing new insights for DMD new drug development.
NETL
NSTL
万方数据
维普
