R6G-PMO45

R6G-PMO45 is a fluorescently labeled antisense oligonucleotide utilized primarily in preclinical research for the treatment of Duchenne Muscular Dystrophy (DMD). The molecule consists of a phosphorodiamidate morpholino oligomer (PMO) sequence specifically engineered to induce the skipping of exon 45 in the dystrophin pre-mRNA, conjugated to the xanthene dye Rhodamine 6G (R6G). This fluorescent tag allows researchers to monitor the systemic delivery, tissue distribution, and intracellular localization of the PMO in animal models, such as the hDMD mouse. By skipping exon 45, the drug aims to restore the genetic reading frame in patients with specific deletions, enabling the production of a truncated but functional dystrophin protein. R6G-PMO45 serves as a critical tool for optimizing delivery vehicles, such as cell-penetrating peptides or nanoparticles, by providing a visual means to assess delivery efficiency across biological barriers.

Other names

Rhodamine 6G-PMO45Rhodamine6G-PMO45Rhodamine-6G-PMO45R6G-conjugated PMO45R-6G-conjugated PMO45R 6G-conjugated PMO45