TY - JOUR
T1 - Historic overview of genetic engineering technologies for human gene therapy
AU - Tamura, Ryota
AU - Toda, Masahiro
N1 - Funding Information:
The authors declare no conflicts of interest associated with this manuscript. This work was supported in part by grants from the Japan Society for the Promotion of Science (JSPS) (18K19622 to M.T.). All authors have registered online Self-reported COI Disclosure Statement Forms through the website for JNS members.
Publisher Copyright:
© 2020 by The Japan Neurosurgical Society.
PY - 2020/10/15
Y1 - 2020/10/15
N2 - 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.
AB - 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.
KW - CRISPR/Cas9
KW - Gene therapy
KW - Genome editing
KW - TALEN
KW - ZFN
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U2 - 10.2176/nmc.ra.2020-0049
DO - 10.2176/nmc.ra.2020-0049
M3 - Review article
C2 - 32908085
AN - SCOPUS:85092687463
SN - 0470-8105
VL - 60
SP - 483
EP - 491
JO - Neurologia Medico-Chirurgica
JF - Neurologia Medico-Chirurgica
IS - 10
ER -
