Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Abstract

The rapid evolution of gene editing technology has markedly improved the outlook for treating genetic diseases. Base editing, recognized as an exceptionally precise genetic modification tool, is emerging as a focus in the realm of genetic disease therapy. We provide a comprehensive overview of the fundamental principles and delivery methods of cytosine base editors (CBE), adenine base editors (ABE), and RNA base editors, with a particular focus on their applications and recent research advances in the treatment of genetic diseases. We have also explored the potential challenges faced by base editing technology in treatment, including aspects such as targeting specificity, safety, and efficacy, and have enumerated a series of possible solutions to propel the clinical translation of base editing technology. In conclusion, this article not only underscores the present state of base editing technology but also envisions its tremendous potential in the future, providing a novel perspective on the treatment of genetic diseases. It underscores the vast potential of base editing technology in the realm of genetic medicine, providing support for the progression of gene medicine and the development of innovative approaches to genetic disease therapy.

Base editing in humanized dystrophic mice

“… 8 , 9 Besides DMD, base editing has been widely pursued to target many genetic or even non-genetic diseases, including hearing loss, blindness, immunodeficiency, cardiovascular diseases, blood disorders, heart failure, liver diseases, neurodegenerative diseases, and viral infection, among others. 10 Excitingly, the first in vivo base-editing clinical trial involving nonviral delivery of ABE targeting PCSK9 has shown promising initial results for the treatment of heterozygous familial hypercholesterolemia. 11 …”