id	author	title	date	pages	extension	mime	words	sentences	flesch	summary	cache	txt
cord-001502-omq0becw	Shabanpoor, Fazel	Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy	2015-01-09		.txt	text/plain	6411	298	53	Studies to date have focused on the delivery of a single SSO conjugated to a CPP, but here we describe the conjugation of two phosphorodiamidate morpholino oligonucleotide (PMO) SSOs to a single CPP for simultaneous delivery and pre-mRNA targeting of two separate genes, exon 23 of the Dmd gene and exon 5 of the Acvr2b gene, in a mouse model of Duchenne muscular dystrophy. Importantly, the cell viability using a bi-specific compound was significantly better than for a mixture of the two individual Pip6a-PMOs. We furthermore assessed the potential of this approach in an in vivo environment through intramuscular administration and demon-Nucleic Acids Research, 2015, Vol. 43, No. 1 31 strated that there were no significant differences in exon skipping activities for both Dmd and Acvr2b targets between bi-specific conjugates (D2 and D3) and a cocktail of the individual P-PMO equivalents.	./cache/cord-001502-omq0becw.txt	./txt/cord-001502-omq0becw.txt