Gene Editing in ANDi: Recent Innovations and Future Prospects

Abstract

Genetic engineering has transformed biomedical research, enabling the creation of animal models that mimic human diseases. Non-human primates (NHPs), due to their genetic similarity to humans, have become key tools for studying complex diseases. The first significant advance in genetic editing of NHPs occurred in 2001 with the birth of ANDi, a Rhesus monkey genetically modified to express the green fluorescent protein gene. Although the study showed limitations, such as low modification efficiency and limited transgene expression, it opened the door for future advances in the genetic modification of primates. Since then, more advanced technologies like CRISPR/Cas9, TALEN, and ZFN have improved the precision and efficiency of genetic editing, enabling the study of various human diseases. These genetically edited NHP models are crucial for translational medicine, the study of neurodegenerative diseases, cancer, and other complex disorders, bringing us closer to effective treatments. Despite technical and ethical challenges, genetic editing in primates remains a critical tool in modern biomedicine.