Genomic editing is going to transform the scientific landscape by accelerating key developments in many disciplines. Although several gene editing approaches with strong clinical impact have been implemented, only a minority of them have direct application to the central nervous system. These limitations are principally due to the intrinsic hurdles in the delivery of the gene editing tools across the brain as well as the limitations in modifying the neuronal genome. Since neurons are post-mitotic cells their genome is reluctant to targeted DNA modifications mediated by the homology-directed-repair (HDR) mechanism. However, innovative methods and approaches have been shown to have the ability to modify (e.g. HITI) or manipulate the expression (e.g. CRISPRa/i) of neuronal genes for potentially treating neurological diseases.
The aim of this Research Topic (research articles and reviews) is to explore recent advances in gene editing applications in neural cells, focusing on the development and/or application of new tools to modify neural cells in vitro and in vivo, from proof-of-concept studies to potential therapeutic approaches.
Genomic editing is going to transform the scientific landscape by accelerating key developments in many disciplines. Although several gene editing approaches with strong clinical impact have been implemented, only a minority of them have direct application to the central nervous system. These limitations are principally due to the intrinsic hurdles in the delivery of the gene editing tools across the brain as well as the limitations in modifying the neuronal genome. Since neurons are post-mitotic cells their genome is reluctant to targeted DNA modifications mediated by the homology-directed-repair (HDR) mechanism. However, innovative methods and approaches have been shown to have the ability to modify (e.g. HITI) or manipulate the expression (e.g. CRISPRa/i) of neuronal genes for potentially treating neurological diseases.
The aim of this Research Topic (research articles and reviews) is to explore recent advances in gene editing applications in neural cells, focusing on the development and/or application of new tools to modify neural cells in vitro and in vivo, from proof-of-concept studies to potential therapeutic approaches.
