The convergences of sophisticated delivery systems, oligonucleotide chemistry and genetic bioinformatics has
significantly enhanced the exploration of antisense oligonucleotides for therapeutic purposes, which has
been reflected in many clinical studies (Nat. Rev. Drug Discov. 11, 125–140, 2012; Annu. Rev. Pharmacol. Toxicol.
50, 259–293, 2010). ASO is a single stranded nucleic acid first introduced in 1978 by Stephenson and Zamecnik
to silence target genes (PNAS, 75, 285-288, 1978). Since then, it has been developed as a potential tool for diverse
application ranging from restoration of protein expression to modification of mutant protein. RNaseH-mediated
degradation or the modulation of splicing of complementary mRNA is the fundamental mode of action in ASObased
therapy. Studies have explored ASO mediated experimental and therapeutic approaches to modulate
biological function at molecular level in both in vitro and in vivo model (Hum. Gene Ther. 26, 475–485, 2015). A
variety of transfection reagents have been used to deliver ASO to human cells. Complexes of ASO with
polycations (e.g., peptides, cationic polymers and dendrimers) are convenient to prepare and offer safe
alternatives for use. The transfection reagents developed by RJH Biosciences have been tested and found
suitable the ASO delivery. This note summarizes the technical experience for ASO delivery for ASO-mediated
modulation of splicing in muscle cells by an independent researcher.