Jordyn Hansel

A CRISPR-based approach to targeting a pathogenic TLR7 gene variant in systemic lupus erythematosus

Started May 30, 2024

Abstract or project description

Systemic lupus erythematosus (SLE) is an autoimmune disease where the body's immune system becomes highly sensitive to non-pathogenic ssRNA, triggering an immune attack on healthy tissue. This incites inflammation, tissue damage, and life-long, adverse side effects. Research has linked over 50 genes to SLE, including the TLR7 gene, which is often overexpressed in individuals with SLE. Recently, a pathogenic variant of the TLR7 gene, known as TLR7-Y264H was demonstrated to oversensitize the toll-like receptors in the cell, resulting in the symptoms of SLE. There is a significant gap in research for SLE treatments, as current options often fail to provide long-term disease control with minimal toxicity. Therefore, this study explores a CRISPR-based approach to rectify this pathogenic variant of TLR7. In light of recent research highlighting the role of TLR7-Y264H mutation’s overexpression in SLE patients, this study investigates whether a CRISPR-based therapeutic approach can effectively dampen SLE symptoms.