HaiFang Yin

TL;DR: It is shown that exosomes—endogenous nano-vesicles that transport RNAs and proteins—can deliver short interfering (si)RNA to the brain in mice, and the therapeutic potential of exosome-mediated siRNA delivery was demonstrated by the strong mRNA and protein knockdown of BACE1, a therapeutic target in Alzheimer's disease, in wild-type mice.

TL;DR: It is shown that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse, with resulting improvement in muscle function.

TL;DR: An investigation of the ability of a model cage, a DNA tetrahedron, to enter live cultured mammalian cells shows that the cages are located in the cytoplasm, a first step toward the use of engineered DNA nanostructures to deliver and control the activity of cargoes within cells.

TL;DR: It is shown that systemic delivery of specific 2OMeAOs, together with the triblock copolymer F127, induced dystrophin expression in all skeletal muscles but not in cardiac muscle of the mdx dystrophic mice, suggesting that a significant therapeutic effect may be achieved by further optimization in dose and regime of administration of antisense oligonucleotide.

TL;DR: Peptide-conjugated AOs have significant potential for systemic correction of the DMD phenotype, with robust sarcolemmal relocalization of the dystrophin-associated protein complex and functional improvement in muscle.