Researchers at Baylor College of Medicine have developed a breakthrough technology for regulating gene expression in gene therapy applications. Published in Nature Biotechnology, the study addresses a long-standing challenge of safely implementing the therapeutic window principle in gene therapy. Current gene regulation systems use foreign regulatory proteins that trigger an immune response, limiting their clinical approval. The Baylor team engineered a system using small molecules to interact with RNA, avoiding immune responses. By placing a switch in the RNA, they can control gene expression levels with FDA-approved doses of tetracycline, offering precise and safe regulation. This technology provides a more flexible and disease-agnostic approach to gene therapy, allowing tailored protein production according to disease stages or patient needs. The compact and easy-to-implement system also holds potential for broader therapeutic applications and laboratory research.
Date:January 2, 2024
Source:Baylor College of Medicine
Liming Luo, Jocelyn Duen-Ya Jea, Yan Wang and Pei-Wen Chao, all at Baylor College of Medicine, also contributed to this work.
This work was supported by an E&M Foundation Pre-Doctoral Fellowship for Biomedical Research, NIH grants (R01EB013584, UM1HG006348, R01DK114356, R01HL130249, P30 CA125123 and S10 RR024574), Biogen SRA, seed fund from Department of Pathology and Immunology at Baylor College of Medicine and CPRIT Core Facility Support Award CPRIT-RP180672.)