Viral-mediated gene therapy in pediatric neurological disorders
Viral-mediated gene therapy in pediatric neurological disorders
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DOI：
10.1007/s12519-022-00669-4
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发表时间：
2023-01
影响因子：
8.7
通讯作者：
Jingbo Peng;Weiwei Zou;Xiao-Lei Wang;Zhiguo Zhang;R. Huo;Li Yang
中科院分区：
文献类型：
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作者：
Jingbo Peng;Weiwei Zou;Xiao-Lei Wang;Zhiguo Zhang;R. Huo;Li Yang
背景由于新一代测序技术的广泛应用，儿童神经科遗传性疾病的分子诊断不再是一个遥不可及的目标。然而，儿童神经遗传性疾病的治疗仍然主要是对症治疗。另一方面，随着治疗性病毒载体的不断发展，基因治疗正在成为临床现实。从这个角度来看，我们写了这篇综述，以说明目前的状态，关于病毒介导的基因治疗在儿童神经系统疾病。数据来源我们搜索数据库，包括PubMed和谷歌学术，使用关键字“腺病毒载体”，“慢病毒载体”，“AAV”的基因治疗，“免疫反应诱导的基因治疗载体”，“管理途径的基因治疗载体，“和“基因治疗”与“NCL”、“SMA”、“DMD”、“先天性肌病”、“MPS”、“脑白质营养不良”或“儿科代谢紊乱”。我们还使用关键词“儿童基因治疗”筛选了ClinicalTrials.gov的数据库，然后用针对神经系统疾病的结果筛选了结果。检索的时间范围是从数据库建立到现在。结果我们介绍了用于儿童神经系统疾病基因治疗的常用病毒载体的特点，并总结了它们的优缺点，每种载体的给药途径，研究进展，以及病毒介导的基因治疗在儿童神经系统疾病中的临床应用现状。介导的基因治疗正处于广泛临床应用的边缘。病毒介导的基因治疗将极大地改变儿童神经系统疾病的治疗模式，许多患有不治之症的儿童将迎来治愈的曙光。然而，必须优化载体以获得更好的安全性和有效性。
BackgroundDue 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 sourcesWe 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” “leukodystrophy,” or “pediatric metabolic disorders”. We also screened the database ofClinicalTrials.govusing 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.ResultsWe 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.ConclusionsViral-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.