Historic overview of genetic engineering technologies for human gene therapy

Ryota Tamura, Masahiro Toda

Research output: Contribution to journalReview articlepeer-review

11 Citations (Scopus)


The concepts of gene therapy were initially introduced during the 1960s. Since the early 1990s, more than 1900 clinical trials have been conducted for the treatment of genetic diseases and cancers mainly using viral vectors. Although a variety of methods have also been performed for the treatment of malignant gliomas, it has been difficult to target invasive glioma cells. To overcome this problem, immortalized neural stem cell (NSC) and a nonlytic, amphotropic retroviral replicating vector (RRV) have attracted attention for gene delivery to invasive glioma. Recently, genome editing technology targeting insertions at site-specific locations has advanced; in particular, the clustered regularly interspaced palindromic repeats/CRISPR-associated-9 (CRISPR/ Cas9) has been developed. Since 2015, more than 30 clinical trials have been conducted using genome editing technologies, and the results have shown the potential to achieve positive patient outcomes. Gene therapy using CRISPR technologies for the treatment of a wide range of diseases is expected to continuously advance well into the future.

Original languageEnglish
Pages (from-to)483-491
Number of pages9
JournalNeurologia medico-chirurgica
Issue number10
Publication statusPublished - 2020 Oct 15


  • CRISPR/Cas9
  • Gene therapy
  • Genome editing
  • ZFN

ASJC Scopus subject areas

  • Surgery
  • Clinical Neurology


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