Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy.It holds out hope of a cure for s...Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy.It holds out hope of a cure for single-gene genetic diseases such as thalassemia,hemophilia,etc.At present,gene therapy is performed in two ways:introducing exogenous genes,and gene editing.A great number of clinical trials of gene therapy in hemophilia have been carried out using viral vectors to introduce foreign genes into target cells.However,the production of neutralizing antibodies following injection and the inability to prepare viral vectors in large quantities limit their application.Although gene-editing methods like CRISPR avoid the above problems,the potential risks of off-target effects are still unknown.More trials and evidence are needed to elucidate the safety and accuracy of gene therapy.This paper will review the bench and clinical work of gene therapy in hemophilia in recent years,and summarize the challenges and prospects of gene therapy,so as to provide directions for future scientific research in this field.展开更多
基金supported by grants from the National Natural Science Foundation of China(No.81300383)the Clinical Research Center of Shandong University(No.2020SDUCRCC015)Young Taishan Scholar Foundation of Shandong Province(No.tsqn202103167).
文摘Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy.It holds out hope of a cure for single-gene genetic diseases such as thalassemia,hemophilia,etc.At present,gene therapy is performed in two ways:introducing exogenous genes,and gene editing.A great number of clinical trials of gene therapy in hemophilia have been carried out using viral vectors to introduce foreign genes into target cells.However,the production of neutralizing antibodies following injection and the inability to prepare viral vectors in large quantities limit their application.Although gene-editing methods like CRISPR avoid the above problems,the potential risks of off-target effects are still unknown.More trials and evidence are needed to elucidate the safety and accuracy of gene therapy.This paper will review the bench and clinical work of gene therapy in hemophilia in recent years,and summarize the challenges and prospects of gene therapy,so as to provide directions for future scientific research in this field.
