Genome Editing in Stem Cells

Research Topic Highlights

This article collection presents novel research utilizing CRISPR-based genome editing and biosensing techniques to elucidate biological processes, enhance biosensing capabilities, and develop targeted therapeutic strategies. Specifically, studies demonstrate precise detection and imaging of DNA sequences at single-locus resolution in live cells using optimized NanoLuc luciferase reporter probes. The collection also highlights the application of CRISPR/Cas9 technology to dissect key developmental processes, such as trophoblast stem cell differentiation and chromatin looping mechanisms regulated by SATB proteins and ELF5. Additionally, research describes the potential therapeutic activation of the PRM1 gene in cancer cells through targeted epigenome editing with CRISPR/dCas9 systems, leading to reduced cellular proliferation. Finally, cell cycle-dependent mechanistic insights into base editing reveal distinct differences between nickase-derived and non-nicking base editors, which could inform precision genome editing strategies. Altogether, these studies underscore the versatility, precision, and efficacy of CRISPR-based platforms across diverse biological and clinical contexts.

Context and Scope

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

Keywords: stem cells, genome editing, CRISPR/Cas, stem cell therapy, diagnosis, CRISPR/Cas9, epigenome editing, base editors, DNA biosensing, NanoLuc luciferase (NLuc), Trophoblast stem cells (TSCs), Syncytialization, Histone modifications, SATB genes, Chromatin looping, ELF5 transcription factor, Protamine 1 (PRM1), Cell cycle dependency, Cancer therapeutic strategies

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.