R6G-PMO53

R6G-PMO53 is a research-grade antisense oligonucleotide used primarily as a fluorescently labeled comparator in preclinical studies for Duchenne muscular dystrophy (DMD). It consists of a phosphorodiamidate morpholino oligomer (PMO) with a sequence designed to skip exon 53 of the dystrophin (DMD) gene, conjugated to the fluorescent dye rhodamine 6G (R6G). The R6G tag facilitates the visualization of tissue distribution and cellular uptake in vivo. In non-human primate studies, R6G-PMO53 has been utilized to establish baseline exon-skipping levels (e.g., approximately 5% after a single dose and 21% after three doses) against which next-generation delivery technologies, such as Endosomal Escape Vehicles (EEVs), are measured. The sequence of the PMO component is typically identical to that of golodirsen.

Other names

Rhodamine 6G-PMO53Rhodamine6G-PMO53Rhodamine-6G-PMO53R6G-conjugated PMO53R-6G-conjugated PMO53R 6G-conjugated PMO53