首页|Viral-mediated gene therapy in pediatric neurological disorders
Viral-mediated gene therapy in pediatric neurological disorders
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Background Due to the broad application of next-generation sequencing,the molecular diagnosis of genetic disorders in pediatric neurology is no longer an unachievable goal.However,treatments for neurological genetic disorders in children remain primarily symptomatic.On the other hand,with the continuous evolution of therapeutic viral vectors,gene therapy is becoming a clinical reality.From this perspective,we wrote this review to illustrate the current state regarding viral-mediated gene therapy in childhood neurological disorders.Data sources We searched databases,including PubMed and Google Scholar,using the keywords"adenovirus vector,""lentivirus vector,"and"AAV"for gene therapy,and"immunoreaction induced by gene therapy vectors,""administration routes of gene therapy vectors,"and"gene therapy"with"NCL,""SMA,""DMD,""congenital myopathy,""MPS""leu-kodystrophy,"or"pediatric metabolic disorders".We also screened the database of ClinicalTrials.gov using the keywords"gene therapy for children"and then filtered the results with the ones aimed at neurological disorders.The time range of the search procedure was from the inception of the databases to the present.Results We presented the characteristics of commonly used viral vectors for gene therapy for pediatric neurological disorders and summarized their merits and drawbacks,the administration routes of each vector,the research progress,and the clinical application status of viral-mediated gene therapy on pediatric neurological disorders.Conclusions Viral-mediated gene therapy is on the brink of broad clinical application.Viral-mediated gene therapy will dramatically change the treatment pattern of childhood neurological disorders,and many children with incurable diseases will meet the dawn of a cure.Nevertheless,the vectors must be optimized for better safety and efficacy.
Jing Peng、Wei-Wei Zou、Xiao-Lei Wang、Zhi-Guo Zhang、Ran Huo、Li Yang
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Department of Pediatrics,Clinical Research Center for Chidren Neurodevelopmental disablities of Hunan Province,Xiangya Hospital,Central South University,Changsha 410008,China
Reproductive Medicine Center,Department of Obstetrics and Gynecology,the First Affiliated Hospital of Anhui Medical University,Hefei,China
State Key Laboratory of Reproductive Medicine,Nanjing Medical University,Nanjing,China
National Key R&D Program of ChinaNational Key R&D Program of ChinaNational Natural Science Foundation of ChinaNational Natural Science Foundation of ChinaKey Research and Development Program of Hunan Province
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