Targeted Editing of Therapeutic Genes Using DNA-Based Transcriptional Activators: Scope and Challenges

Abstract

In recent years, there has been an increasing interest among the clinicians to devise genetic knowledge-based therapeutic strategies to treat complex diseases. Since targeted treatments ensure better drug efficacy and fewer long-term side effects, development of tailor-made drugs is of rising demand. Through modern biological and analytical techniques, we could now predict the transcription factors associated with disorders and design chemical and/or biological tools to reset the transcriptional machinery of the diseased cell and restore them back to healthy state. However, therapeutic targeting of the defected genes in the body is not straightforward owing to the critical influence of the dynamic epigenome. In this chapter, we give a detailed overview of the customizable artificial transcription activators and therapeutically significant transcription factors and suggest the need to gain inspiration from the coordinated chromatin modifications observed in natural cellular environment and design targeting transcriptional activators with epigenetic activity.