Research Topic

Advanced Gene Editing Tools for Basic Research and Disease Therapy

About this Research Topic

Gene editing is capable of editing DNA sequence in vivo, holding tremendous potential in basic research and treatment of disease. The rapid development of gene editing tools, particularly tools adapted from CRISPR/Cas system, has revolutionized both basic and translational research. However, many issues remain unaddressed, such as in vivo delivery, off-target effects and imprecise on-target site editing. In vivo delivery of gene editing tools can take advantage of viral vectors, cationic lipids, cationic polymer or lipid nanoparticles. Highly efficient and specific delivery has not yet been achieved in different tissues, impeding the clinical translation of gene editing. Off-target effects can induce unwanted mutations, representing another roadblock for gene editing therapy. In addition, persisted expression of gene editing tools can lead to more off-target editing. Besides off-target effects, editing at on-target sites can result in unintended mutations at the target sites, such as large deletion, genomic rearrangements, and by-stander mutation effects etc.

Therefore, more efforts should be devoted to improving in vivo delivery, reducing the off-target effects, and eliminating unintended mutations. Cancer treatment is often subject to the development of drug resistance, highlighting the need to develop new therapeutics. Synthetic lethal and syntheic viable screens by CRISPR library could not only decipher cancer biology but also identify novel treatment targets. Moreover, tumor growth could be reduced by in vivo gene editing. In addition, immune cells could be engineered by gene editing to improve the efficacy of cancer immunotherapy. Till now, gene editing tools have shown great promise in treating cancer, and several clinical trails are underway.

In this Research Topic, we would like to collect manuscripts related to the following issues:

1. Developments of new methods for in vivo delivery of gene editing tools.
2. Developments of new methods to detect or reduce the off-target effects of gene editing tools.
3. Developments of new controllable gene editing tools for in vivo gene editing therapy.
4. Controlling gene editing outcome through manipulating DNA repair mechanism.
5. Application of gene editing in cancer research and treatment.

Manuscripts types include original research articles, brief research reports and review articles.


Keywords: controllable gene editing, in vivo delivery, off-target effects, DNA repair, cancer research, cancer treatment


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.

Gene editing is capable of editing DNA sequence in vivo, holding tremendous potential in basic research and treatment of disease. The rapid development of gene editing tools, particularly tools adapted from CRISPR/Cas system, has revolutionized both basic and translational research. However, many issues remain unaddressed, such as in vivo delivery, off-target effects and imprecise on-target site editing. In vivo delivery of gene editing tools can take advantage of viral vectors, cationic lipids, cationic polymer or lipid nanoparticles. Highly efficient and specific delivery has not yet been achieved in different tissues, impeding the clinical translation of gene editing. Off-target effects can induce unwanted mutations, representing another roadblock for gene editing therapy. In addition, persisted expression of gene editing tools can lead to more off-target editing. Besides off-target effects, editing at on-target sites can result in unintended mutations at the target sites, such as large deletion, genomic rearrangements, and by-stander mutation effects etc.

Therefore, more efforts should be devoted to improving in vivo delivery, reducing the off-target effects, and eliminating unintended mutations. Cancer treatment is often subject to the development of drug resistance, highlighting the need to develop new therapeutics. Synthetic lethal and syntheic viable screens by CRISPR library could not only decipher cancer biology but also identify novel treatment targets. Moreover, tumor growth could be reduced by in vivo gene editing. In addition, immune cells could be engineered by gene editing to improve the efficacy of cancer immunotherapy. Till now, gene editing tools have shown great promise in treating cancer, and several clinical trails are underway.

In this Research Topic, we would like to collect manuscripts related to the following issues:

1. Developments of new methods for in vivo delivery of gene editing tools.
2. Developments of new methods to detect or reduce the off-target effects of gene editing tools.
3. Developments of new controllable gene editing tools for in vivo gene editing therapy.
4. Controlling gene editing outcome through manipulating DNA repair mechanism.
5. Application of gene editing in cancer research and treatment.

Manuscripts types include original research articles, brief research reports and review articles.


Keywords: controllable gene editing, in vivo delivery, off-target effects, DNA repair, cancer research, cancer treatment


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

15 August 2021 Abstract
13 December 2021 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

15 August 2021 Abstract
13 December 2021 Manuscript

Participating Journals

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

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