Bioinformatics: Tissue-specific gene therapy constructs using snATAC-seq and scRNA-seq
Our team collaborated with a major pharmaceutical client to design tissue-specific plasmid constructs, enhancing the precision of gene therapy payloads for targeted tissue delivery. By employing four distinct strategies, we identified key core promoter regions and relevant enhancer elements essential for maximizing tissue-specific expression. These optimized constructs support more effective and precise gene therapy, advancing the potential of targeted therapeutic applications.
Strategy 1
Net signal intensity :
K2 - Max (Lu.2,Li.2, H2)
Net signal intensity :
K1 - Max (Lu.1,Li.1, H1)
Net signal intensity :
K3 - Max (Lu.3,Li.3, H3)
DHS - DNAse Hypersensitivity Site, Lu - Lung, Li - Liver, H - Heart
Strategy 2
Strategy 3
Strategy 4
PCT: Proximal Convoluted Tubule, PST: Proximal Straight Tubule, PTVCAM1: Proximal Tubule with enhanced expression of VCAM1
TTA: Spacer 3-nt sequences