In the medical and pharmaceutical fields, stem cells and programmable nuclease platforms provide a new viewpoint by enabling the development of in vivo or in vitro disease models, accurate gene editing, precise off-target or diagnostic tool detection, and functional improvements in therapeutic genes. Unlike their wild-type counterparts, genetically engineered stem cells offer many advantages including enhanced secretion of therapeutic proteins, enhanced homing, and an increased rate of survival following transplantation. Among the technologies used for improving cell functions are a wide range of genome editing platforms, including CRISPR/Cas, ZFN, and TALEN. The CRISPR/Cas system is one of the most widely accepted systems due to its ability to precisely edit DNA and RNA, as well as its ease of handling and construction. Additionally, it is currently being expanded with its new orthologues that make it possible to edit RNA and DNA at high accuracy and precision.
Despite the feasibility of using genome editing tools to correct genetic mutations, there are some limitations, such as off-target effects and safety concerns, which need to be explored when considering the clinical application of these techniques. Significant efforts have been made to overcome these limitations.
This Research Topic encourages the submissions of articles that focus on the following topics, but are not limited to:
• Genome editing techniques and methods of advancement
• Wide range of applications of genome engineering platforms
• Development of safe and efficient gene-edited stem cell therapies
In the medical and pharmaceutical fields, stem cells and programmable nuclease platforms provide a new viewpoint by enabling the development of in vivo or in vitro disease models, accurate gene editing, precise off-target or diagnostic tool detection, and functional improvements in therapeutic genes. Unlike their wild-type counterparts, genetically engineered stem cells offer many advantages including enhanced secretion of therapeutic proteins, enhanced homing, and an increased rate of survival following transplantation. Among the technologies used for improving cell functions are a wide range of genome editing platforms, including CRISPR/Cas, ZFN, and TALEN. The CRISPR/Cas system is one of the most widely accepted systems due to its ability to precisely edit DNA and RNA, as well as its ease of handling and construction. Additionally, it is currently being expanded with its new orthologues that make it possible to edit RNA and DNA at high accuracy and precision.
Despite the feasibility of using genome editing tools to correct genetic mutations, there are some limitations, such as off-target effects and safety concerns, which need to be explored when considering the clinical application of these techniques. Significant efforts have been made to overcome these limitations.
This Research Topic encourages the submissions of articles that focus on the following topics, but are not limited to:
• Genome editing techniques and methods of advancement
• Wide range of applications of genome engineering platforms
• Development of safe and efficient gene-edited stem cell therapies