Advancing Approaches of Gene Correction in Therapeutic Applications: Exploring Front-Line Technologies Beyond CRISPR-Cas9

Abstract

Gene correction technologies have revolutionized the field of genetic medicine, offering promising avenues for the treatment of various genetic diseases. This manuscript delves into the advancements in gene correction approaches beyond CRISPR-Cas9, shedding light on the challenges and opportunities in this rapidly evolving field. The review encompasses a comprehensive analysis of nuclease and nickase genome editing methods, highlighting the complexities encountered in translating these techniques to clinical settings. Furthermore, the exploration of base editors for correcting point mutations in monogenic diseases underscores the potential of precise genetic engineering tools in addressing a wide range of disorders. The integration of transcription activator-like effector domain (TALE) base editors and the development of piggyPrime for high-efficiency prime editing showcase innovative strategies for multiplex gene engineering and genomic integration, respectively. Additionally, the study on off-target editing outcomes in non-human primate embryos emphasizes the critical need for thorough sequencing-based techniques to evaluate editing outcomes and ensure the safety of gene editing approaches. Overall, this communication underscores the importance of advancing gene correction technologies to bring effective therapeutic interventions closer to patients in need, while also addressing the challenges associated with unintended editing effects and safety considerations.

Cell Mol Med Res. 2024;000(000):000-000
doi: https://doi.org/10.14740/cmmr55