首页|Gene therapy for monogenic disorders:challenges,strategies,and perspectives
Gene therapy for monogenic disorders:challenges,strategies,and perspectives
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Monogenic disorders refer to a group of human diseases caused by mutations in single genes.While disease-modifying therapies have offered some relief from symptoms and delayed progression for some monogenic diseases,most of these diseases still lack effective treatments.In recent decades,gene therapy has emerged as a promising therapeutic strategy for genetic disorders.Researchers have developed various gene manipulation tools and gene delivery systems to treat monogenic diseases.Despite this progress,concerns about inefficient delivery,persistent expression,immunogenicity,toxicity,capacity limitation,genomic integration,and limited tissue specificity still need to be addressed.This review gives an overview of commonly used gene therapy and delivery tools,along with the challenges they face and potential strategies to counter them.
Department of Medical Genetics and Center for Rare Diseases,Second Affiliated Hospital,Zhejiang University School of Medicine,Hangzhou,Zhejiang 310009,China
Department of Neurology,Second Affiliated Hospital,Zhejiang University School of Medicine,Hangzhou,Zhejiang 310009,China
Key Laboratory of Medical Neurobiology of Zhejiang Province,Hangzhou,Zhejiang 310009,China
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