Extended Data Fig. 1: PPMO structure.
From: A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
SRP-2001 is a peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) comprising a cell-penetrating peptide (CPP) covalently linked to a phosphorodiamidate morpholino oligomer (PMO). The PMO contains 25 morpholino subunits each bearing a nucleobase forming the sequence 5’-GAGGAGATGGGTCCACCCACCTGGG-3’. The conjugated peptide comprises the amino acid sequence R6G, where R is arginine and G is glycine. The glycine residue covalently links the CPP to the PMO by an amide bound to the 3’ end of the PMO. Mass spectrometric characterization was performed to confirm the structure and sequence of SRP-2001.
