SHENZHEN Hemogen Therapeutic Co., Ltd.

The regulatory element, that drives the target gene expression, has a decisive impact on the efficacy and safety for lentiviral vector (LV) based on gene therapy. Through the comprehensive analysis of the high-throughput sequencing data of the hematopoietic system, our OmiRES platform first mines the specifically expressed genes and their regulatory elements for each cell group of interest. The identified elements are coupled with different tag sequences (Barcodes), constituting the element library, enable us to high-throughput evaluate their transcription efficiency and to find optimal elements for gene-therapy in certain type of cells. The use of optimized elements can effectively enhance the expression efficiency and specificity of effector genes in target cells, and will improve the safety and efficacy of LV based gene therapy.