In the last few years, the CRISPR-Cas9 gene editing technology has revolutionized the biomedical field, having originated a 2020 Nobel Prize. The repurposing of the bacterial immune system now provides a ground-breaking tool for targeted gene regulation. Since CRISPR-Cas9 can target virtually any genomic sequence (by tailored expression of ~20 bp guide RNA (sgRNA)), it opens unprecedented opportunities for basic and clinical research. Several clinical trials are currently underway, some of which already proved the safety and efficacy of this system. Recent progress on CRISPR-directed gene therapies has opened a new era for medicine. However, several challenges remain.

In this Research Topic, recent progress and future perspectives of this simple but versatile tool will be explored, not disregarding the major concerns, namely environmental implications, technical limitations and ethical issues. We encourage the submission of manuscripts spanning a wide range of topics, ranging from basic mechanisms of genome editing to clinical translation and therapeutic application.

The original CRISPR publication was in 2012, and the question remains a decade later on how this technology is progressing and impacting basic science and clinical application. We will cover topics ranging from novel CRISPR tool design, pre-clinical application development, clinical application, FDA regulation, and ethical considerations of CRISPR gene editing.

We welcome Original Research, Brief Research Reports, Reviews, Systematic Reviews, Mini-Reviews, Policy and Practice Reviews, Policy Briefs, Methods, Perspectives, Hypothesis and Theories, and Opinion articles including but not limited to the following topics:

• Efficient, safe, and specific CRISPR delivery
• Base editing
• Epigenome Editing/CRISPRi/CRISPRa
• CRISPR-based diagnostics and precision medicine
• Methods to improve system efficiency and minimize off-target effects
• CRISPR as a tool for disease modeling
• Ethical and legal challenges
• Environmental hazards of the technology
• Preclinical studies

Clinical studies and manuscripts describing purely bioinformatic analyses of existing databases without significant experimental validation will not be accepted to this collection, as they are outside the scope of the Preclinical Cell and Gene Therapy section, of which this Research Topic is a part.

Dr. Joana Caldeira is one of the co-founders of the startup FETALDISC, Lda and inventor
of the filed patent WO2021118379A1.
Dr. Robert Bowles holds a US patent WO-2016205688-A3.

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Frontiers in Bioengineering and Biotechnology

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