Viral-mediated gene therapy in pediatric neurological disorders

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""leukodystrophy,"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.

Imprinting methylation and assisted reproductive technology

Assisted reproductive technology （ART） is an important treatment for infertile people of reproductive age and is also known as fertility treatment. The processes of ART involves the isolation, handling and culture of early embryos, which may result in alterations in genomic methylation at specific loci and influence the proper establishment and maintenance of genomic imprints. Recent studies have identified an increased incidence of imprinting disorders via ART. In this article, we reviewed that the ART may be prone to induction of imprinting methylation errors during embryonic development. Further studies are necessary to elucidate the safety of ART in this field.