Research Topic

Genome Engineering in Induced Pluripotent Stem Cells: Tools and Modeling the Central Nervous System and Neurological Diseases

About this Research Topic

Genome editing is a rapidly developing technology used to model disease (cellular and organoid) by engineering causative mutations such as SNPs, large deletions, or duplications in induced pluripotent stem cells (iPSCs) and to establish genome-wide approaches to understand the central nervous system and neurological diseases. The use of genome engineering on patient iPSCs has revolutionized the studies directed at understanding the central nervous system and diseases of the brain by creating true isogenic controls as well as allelic series containing different causative variants with the same genetic background. Next generation sequencing of human patient-derived clinical samples continues to identify a multitude of mutations correlated with neurological diseases and the use of genome engineering will allow us to better understand whether these are causative factors in disease development.

The aim of the Research Topic (research articles and reviews) is to provide the research community new engineered models of disease, new tools for rapid genome editing and approaches that utilize CRISPR/Cas9 or other DNA-binding domain technology. Applications of both gene-focused and gene-wide approaches that further our understanding of the central nervous system and diseases of the brain will be considered. Methods for establishment of efficient and improved CRISPR/Cas9 engineering as well as providing standardization in the field are also the aim of this Research Topic.


Keywords: CRISPR, neurological disease modeling, genome editing


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.

Genome editing is a rapidly developing technology used to model disease (cellular and organoid) by engineering causative mutations such as SNPs, large deletions, or duplications in induced pluripotent stem cells (iPSCs) and to establish genome-wide approaches to understand the central nervous system and neurological diseases. The use of genome engineering on patient iPSCs has revolutionized the studies directed at understanding the central nervous system and diseases of the brain by creating true isogenic controls as well as allelic series containing different causative variants with the same genetic background. Next generation sequencing of human patient-derived clinical samples continues to identify a multitude of mutations correlated with neurological diseases and the use of genome engineering will allow us to better understand whether these are causative factors in disease development.

The aim of the Research Topic (research articles and reviews) is to provide the research community new engineered models of disease, new tools for rapid genome editing and approaches that utilize CRISPR/Cas9 or other DNA-binding domain technology. Applications of both gene-focused and gene-wide approaches that further our understanding of the central nervous system and diseases of the brain will be considered. Methods for establishment of efficient and improved CRISPR/Cas9 engineering as well as providing standardization in the field are also the aim of this Research Topic.


Keywords: CRISPR, neurological disease modeling, genome editing


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.

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Submission Deadlines

31 October 2020 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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Topic Editors

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Submission Deadlines

31 October 2020 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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