Compact TSPO promoter fragments enable efficient transgene delivery to microglia, overcoming vector capacity limits for neurological therapies.
Using cultured XEN cells allows in vitro genetic screening, eliminating laborious animal breeding and reducing mosaicism.
Combining a histone stem-loop structure with a polyadenylation signal stabilizes mRNA against degradation while boosting protein expression levels.
Integrating gene transfer vectors incorporate multiple copies of CTCF or CTF binding sites as genetic insulator elements to prevent regulatory cross-talk.
Self-targeting DNA vectors use integrated binding motifs to bypass nuclear import bottlenecks, enhancing transfection while eliminating viral immunogenicity.
Lentiviral vectors deliver the SLC25A38 gene to hematopoietic stem cells.
Chimeric BaEV-MLV envelope glycoproteins resolve complement sensitivity and quiescent cell resistance in hematopoietic stem cell gene therapy.
Group I intron self-splicing merges transcription and circularization into one reaction, eliminating DNase I treatment and separate purification steps.
Removing non-paired donor splice sites in antibody variable domains increases IgG1 expression yields.
Combining diaphragm and cardiac muscle-specific regulatory elements drives robust transgene expression while reducing vector doses and associated toxicity.
Native G6PC promoter and synthetic intron in AAV vectors correct hepatic glucose-6-phosphatase deficiency and normalize metabolic parameters.
SYNP88 promoter combines viral core strength with cellular enhancer specificity to achieve high-level gene expression in retinal ganglion cells.