Gene Editing: New Frontiers to Fight Genetic Diseases

Genome editing is revolutionizing the medical landscape by offering new therapeutic possibilities for genetic diseases, which collectively affect hundreds of millions of people worldwide. The field has rapidly evolved from early CRISPR–Cas9 systems, which depend on DNA double-strand break repair, to sophisticated technologies such as base and prime editing that allow for more accurate, efficient, and predictable genomic modifications. Recent breakthroughs include base editing’s capacity for single-nucleotide conversion without cleaving the DNA, and prime editing’s proficiency in introducing diverse genetic changes, including point mutations, insertions, and deletions. Notably, these advances have progressed from laboratory models to early clinical trials, with base editing therapies for transthyretin amyloidosis and prime editing’s increasing target scope reflecting the pace of translation from bench to bedside.These innovations have begun to reshape clinical approaches to treating genetic conditions. CRISPR-based therapies are now achieving landmark outcomes, including the first regulatory approvals for sickle cell disease and β-thalassemia. Improved delivery platforms - for example, lipid nanoparticles and next-generation viral vectors—have broadened the application of genome editing in vivo. Nonetheless, significant challenges persist. These include editing efficiency disparities among tissues, risk of off-target and bystander effects, immune reactions to editing machinery, and the need for long-term safety data. Latest studies have highlighted the necessity of optimizing specificity and minimizing unintended consequences, driving development toward more refined editing tools, such as engineered CRISPR proteins and RNA-mediated systems.This Research Topic aims to gather and advance knowledge on the newest frontiers in genome editing as a therapeutic modality for genetic diseases. We seek to address pivotal questions concerning the efficacy, safety, and translational readiness of emerging gene editing platforms. Key objectives include evaluating the molecular mechanisms underpinning precision editing, comparing clinical performance across technologies, exploring innovative delivery routes, and critically examining the societal and ethical framings of therapeutic genome modification.To comprehensively address the challenges and opportunities in gene editing for genetic disease, we welcome articles that focus on both the molecular underpinnings and translational hurdles in this field, without extending to areas unrelated to therapeutic genome modification. We invite contributions on, but not limited to, the following themes:o Advances in gene targeting and correction strategies for genetic diseaseso Clinical progress, challenges, and innovations in CRISPR-based therapieso Applications and refinements of base editing, prime editing, and next-generation editing platformso Developments in delivery methods, including lipid nanoparticles and engineered vectors, for enhancing in vivo editingo Assessment of safety, specificity, long-term outcomes, and immune considerations in genome editingo Ethical, regulatory, and societal implications of gene editing for therapeutic useWe invite submissions in the form of original research articles, reviews, and commentaries that deliver fresh perspectives or data in this rapidly evolving area. Topic editors: Elena Paccosi, Luca Proietti De Santis, Silvia Filippi Source: https://www.frontiersin.org/research-topics/80731/gene-editing-new-frontiers-to-fight-genetic-diseases