Regulation of Gene Expression, a New Therapeutic Approach
Human diseases often arise from changes in gene expression levels. Moreover, altering the expression levels of specific genes can provide therapeutic benefits for various diseases. Systems combining nuclease-deficient gene-editing proteins with transcriptional modulators to target gene regulatory elements have emerged, offering a means to modify gene expression for therapeutic gain. These systems have the potential to provide therapeutic benefits by modifying gene expression. Several systems are already being used in clinical settings, and many more are under development. This review evaluates the therapeutic potential, delivery, and clinical challenges associated with these systems.
Numerous human diseases are caused by changes in gene expression levels. In addition, changing the expression levels of specific genes can lead to therapeutic benefits for several diseases. Systems using nuclease-deficient gene-editing proteins fused to transcriptional modulators that target gene regulatory elements have emerged as powerful, programmable, and customizable systems to modify gene expression for therapeutic benefits. Several of these systems have already been used in the clinic, and many more are under development. Here, we review these emerging technologies and assess their therapeutic potential, their delivery, and related challenges in the clinic.
This research is scientifically and socially significant because it offers a new approach to treating diseases. The potential to regulate gene expression to treat diseases is substantial. Furthermore, the development of this technology could have a significant impact on human health.