PLGA-Nanoparticles for Intracellular Delivery of the CRISPR-Complex to Elevate Fetal Globin Expression in Erythroid Cells

Globin gene expression in erythroid human fetal liver cells.

We measured steady-state levels of the human globin mRNAs in liver samples from several mid-gestational fetuses. RNA from the epsilon, gamma, beta, zeta, theta, and alpha globin genes were present in fetal liver samples isolated from 10-25-wk embryos. The abundance of all human globin mRNAs declined in older fetuses, presumably because of a gradual reduction in the proportion of erythroid precu...

The role of the polycomb complex in silencing α-globin gene expression in non-erythroid cells

Although much is known about globin gene activation in erythroid cells, relatively little is known about how these genes are silenced in non-erythroid tissues. Here we show that the human αand β-gobin genes are silenced by fundamentally different mechanisms. The α-genes, which are surrounded by widely expressed genes in a gene dense region of the genome, are silenced very early in development v...

Recombinant erythroid Kruppel-like factor fused to GATA1 upregulates globin expression in erythroid cells

PLGA Nanoparticles for Ultrasound-Mediated Gene Delivery to Solid Tumors

This paper focuses on novel approaches in the field of nanotechnology-based carriers utilizing ultrasound stimuli as a means to spatially target gene delivery in vivo, using nanoparticles made with either poly(lactic-co-glycolic acid) (PLGA) or other polymers. We specifically discuss the potential for gene delivery by particles that are echogenic (amenable to destruction by ultrasound) composed...

conjugating plga nanoparticles with hsa for delivery to cancerous tissue