Viral-mediated gene therapy in pediatric neurological disorders
作者机构：Department of PediatricsClinical Research Center for Chidren Neurodevelopmental disablities of Hunan ProvinceXiangya HospitalCentral South UniversityChangsha 410008China Reproductive Medicine CenterDepartment of Obstetrics and Gynecologythe First Affiliated Hospital of Anhui Medical UniversityHefeiChina State Key Laboratory of Reproductive MedicineNanjing Medical UniversityNanjingChina
出 版 物：《World Journal of Pediatrics》 (世界儿科杂志（英文版）)
年 卷 期：2024年第20卷第6期
页 面：533-555页
核心收录：
学科分类：1004[医学-公共卫生与预防医学(可授医学、理学学位)] 1002[医学-临床医学] 100204[医学-神经病学] 10[医学]
基 金：supported by the National Key R&D Program of China(Grant number:2021YFC2700903,2021YFC2700901) the National Natural Science Foundation of China(Grant numbers:82001449 and 82001635) the Key Research and Development Program of Hunan Province(Grant number:2022SK2036)
主 题：Gene therapy Neurological disorders Pediatrics Viral vectors
摘 要：Background Due to the broad application of next-generation sequencing,the molecular diagnosis of genetic disorders in pediatric neurology is no longer an unachievable ***,treatments for neurological genetic disorders in children remain primarily *** the other hand,with the continuous evolution of therapeutic viral vectors,gene therapy is becoming a clinical *** this perspective,we wrote this review to illustrate the current state regarding viral-mediated gene therapy in childhood neurological *** sources We searched databases,including PubMed and Google Scholar,using the keywordsadenovirus vector,lentivirus vector,andAAvfor gene therapy,andimmunoreaction induced by gene therapy vectors,administration routes of gene therapy vectors,andgene therapywithNCL,SMA,DMD,congenital myopathy,MPSleukodystrophy,orpediatric metabolic disorders.We also screened the database of *** using the keywordsgene therapy for childrenand then filtered the results with the ones aimed at neurological *** time range of the search procedure was from the inception of the databases to the *** 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 *** Viral-mediated gene therapy is on the brink of broad clinical ***-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 ***,the vectors must be optimized for better safety and efficacy.
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