Gene editing is commonly split into two major categories based on the engaged repair pathways: non-homologous end-joining and homology-directed repair (HDR) a.k.a. homologous recombination. The former is mainly used for gene knockout whereas the latter can be exploited for precise DNA replacement or addition. The HDR pathway has traditionally been regarded as the most inefficient of the two, but recent breakthroughs have enables very high frequencies of HDR even in ‘difficult-to-engineer’ primary cells. New additions to the toolbox, such as base and prime editing, have opened up possibilities of exploiting other repair mechanisms to introduce precise changes to the genome. However, multiple challenges and unanswered questions remain for the full application of these CRISPR-based tools and other site-specific engineered nucleases to cure human disease.
In this Research Topic, we will feature recent findings within genome editing technologies that focus on repair, addition, and replacement outcomes in human cells or relevant animal models. We welcome the submission of articles that aid the basic understanding of precise genome editing and facilitate the clinical translation and therapeutic implementation of these tools in blood disorders or other monogenic diseases.
We encourage submissions in the form of Original Research, Methods, Reviews, Mini Review, Opinion, and Hypothesis and Theory.
Suitable topics include, but are not limited to:
· HDR-based gene editing using CRISPR and other engineered nuclease systems
· Base and Prime Editing tools
· Enhancement/optimization of current approaches to precise gene editing
· Understanding of HDR mechanisms in the context of gene correction and gene addition.
· Evaluation of HDR gene editing outcomes
· Off-target donor DNA integration
· Challenges in cDNA integration approaches
· Viral vs non-viral donor DNA delivery
· dsDNA vs ssODNs donor DNA applications
· Ex vivo and in vivo therapeutic DNA replacement strategies
Gene editing is commonly split into two major categories based on the engaged repair pathways: non-homologous end-joining and homology-directed repair (HDR) a.k.a. homologous recombination. The former is mainly used for gene knockout whereas the latter can be exploited for precise DNA replacement or addition. The HDR pathway has traditionally been regarded as the most inefficient of the two, but recent breakthroughs have enables very high frequencies of HDR even in ‘difficult-to-engineer’ primary cells. New additions to the toolbox, such as base and prime editing, have opened up possibilities of exploiting other repair mechanisms to introduce precise changes to the genome. However, multiple challenges and unanswered questions remain for the full application of these CRISPR-based tools and other site-specific engineered nucleases to cure human disease.
In this Research Topic, we will feature recent findings within genome editing technologies that focus on repair, addition, and replacement outcomes in human cells or relevant animal models. We welcome the submission of articles that aid the basic understanding of precise genome editing and facilitate the clinical translation and therapeutic implementation of these tools in blood disorders or other monogenic diseases.
We encourage submissions in the form of Original Research, Methods, Reviews, Mini Review, Opinion, and Hypothesis and Theory.
Suitable topics include, but are not limited to:
· HDR-based gene editing using CRISPR and other engineered nuclease systems
· Base and Prime Editing tools
· Enhancement/optimization of current approaches to precise gene editing
· Understanding of HDR mechanisms in the context of gene correction and gene addition.
· Evaluation of HDR gene editing outcomes
· Off-target donor DNA integration
· Challenges in cDNA integration approaches
· Viral vs non-viral donor DNA delivery
· dsDNA vs ssODNs donor DNA applications
· Ex vivo and in vivo therapeutic DNA replacement strategies