Essential stem cell maintenance genes, including Pax7, a transcription factor fundamental to preserving the high regenerative capacity of muscles, is induced via targeting DNA secondary structures.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
Modulate oxygen-releasing erythroid progenitor cells (EPCs) to promote low oxygen conditions (hypoxia) and enhance bone fracture repair.
Ten candidate serum and synovial protein levels provide early indication of joint impairment in lysosomal storage disorders.
A library of bi-phosphonate ionizable lipids and LNP formulations to facilitate mRNA delivery to the bone microenvironment.
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.
Simplified and reproducible two-step differentiation method optimized for efficient differentiation of human-induced pluripotent stem cells into retinal progenitor cells and functional retinal ganglion cells
Improved cartilage repair via injectable modified polymers that target damaged tissues and enhance cellular response