Gene Editing: New Frontiers to Fight Genetic Diseases

The goal of genome editing technology is to develop strategies for the correction of those mutations that are responsible of a plethora of genetic diseases that collectively affect hundreds of millions of people. The main part of known genetic diseases require a precise correction of the target gene. This is why CRISPR–Cas9, which edits DNA by activating DNA double-strand break repair pathways, is not always sufficient for gene therapy applications. Along this line, two new types of gene editing has been developed to produce precise changes in genes: base editing, which can change a single DNA letter, and prime editing, which is able to delete or repair long lengths of DNA or insert DNA in order to repair potentially deleterious mutations. As an example, base editing in animal models has been shown to be able to modify the mutant gene that causes progeria and reduce the disease’s effects.

In the context of genetic diseases, genome editing technology represents a promising approach to correct the mutation and treat the resulting disease. Recent advances in CRISPR-based genome editing can allow to direct the precise and specific modification of a certain gene. However, there are many limitations; as an example, prime editing shows a low editing efficiency. Concerns arise also regarding the safety of genome editing technology. Further studies are definitely mandatory to ameliorate the efficacy and safety of this technology, thus enabling this kind of approach to the development of innovative and specific therapies.

For this Research Topic, we invite contributions exploring the scope of this technology in counteracting genetic diseases. Specific themes include, but are not limited to, the precise modification of genetic disease-related genes, the new types of gene editing strategies, and the development of innovative therapies taking advantage of these technologies.

We are interested in original research articles, review papers and commentaries which give new insights on this promising and interesting field:

• Gene Targeting in Genetic diseases Research
• CRISPR-Cas9 in Fighting Genetic Diseases: pros and cons
• Base and Prime Editing to correct pathogenic mutations
• Recent advances in the treatment of a particular genetic disease by genome editing technology
• Genome Editing: how far can we go? Ethical considerations on this new technology in genetic disease treatment

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Editorial
• FAIR² Data
• FAIR² DATA Direct Submission
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Editorial
• FAIR² Data
• FAIR² DATA Direct Submission
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Policy and Practice Reviews
• Policy Brief
• Review
• Systematic Review

Keywords: genome editing, genetic diseases, editing technologies, base editing, prime 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.