Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes

Simpson, R.J., Jensen, S.S. & Lim, J.W. Proteomic profiling of exosomes: current perspectives. Proteomics 8, 4083–4099 (2008).

Schorey, J.S. & Bhatnagar, S. Exosome function: from tumor immunology to pathogen biology. Traffic 9, 871–881 (2008).

Kumar, P. et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 448, 39–43 (2007).

Seow, Y. & Wood, M.J. Biological gene delivery vehicles: beyond viral vectors. Mol. Ther. 17, 767–777 (2009).

Valadi, H. et al. Exosome-mediated transfer of mRNAs and microRNAs is a novel mechanism of genetic exchange between cells. Nat. Cell Biol. 9, 654–659 (2007).

Skog, J. et al. Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers. Nat. Cell Biol. 10, 1470–1476 (2008).

Quah, B.J. & O'Neill, H.C. The immunogenicity of dendritic cell-derived exosomes. Blood Cells Mol. Dis. 35, 94–110 (2005).

Inaba, K. et al. Generation of large numbers of dendritic cells from mouse bone marrow cultures supplemented with granulocyte/macrophage colony-stimulating factor. J. Exp. Med. 176, 1693–1702 (1992).

Théry, C., Amigorena, S., Raposo, G. & Clayton, A. Isolation and characterization of exosomes from cell culture supernatants and biological fluids. in Current Protocols Cell Biology, chapter 3, 3.22 (John Wiley, 2006).

Simhadri, V.R. et al. Dendritic cells release HLA-B-associated transcript-3 positive exosomes to regulate natural killer function. PLoS ONE 3, e3377 (2008).

Flint, P.W., Li, Z.B., Lehar, M., Saito, K. & Pai, S.I. Laryngeal muscle surface receptors identified using random phage library. Laryngoscope 115, 1930–1937 (2005).

Larson, S.D., Jackson, L.N., Chen, L.A., Rychahou, P.G. & Evers, B.M. Effectiveness of siRNA uptake in target tissues by various delivery methods. Surgery 142, 262–269 (2007).

Yin, H. et al. A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum. Mol. Genet. 18, 4405–4414 (2009).

Nunes, F.A., Furth, E.E., Wilson, J.M. & Raper, S.E. Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: safety of readministration. Hum. Gene Ther. 10, 2515–2526 (1999).

Gonzalez, S.C. et al. Readministration of adenoviral gene delivery to dopamine neurons. Neuroreport 18, 1609–1614 (2007).

Laird, F.M. et al. BACE1, a major determinant of selective vulnerability of the brain to amyloid-beta amyloidogenesis, is essential for cognitive, emotional, and synaptic functions. J. Neurosci. 25, 11693–11709 (2005).

Singer, O. et al. Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat. Neurosci. 8, 1343–1349 (2005).

Nishitomi, K. et al. BACE1 inhibition reduces endogenous Abeta and alters APP processing in wild-type mice. J. Neurochem. 99, 1555–1563 (2006).

Aguzzi, A. et al. Protein aggregation diseases: pathogenicity and therapeutic perspectives. Nat. Rev. Drug Discov. 9, 237–248 (2010).

Kao, S.C., Krichevsky, A.M., Kosik, K.S. & Tsai, L.H. BACE1 suppression by RNA interference in primary cortical neurons. J. Biol. Chem. 279, 1942–1949 (2004).

Mosmann, T. Rapid colorimetric assay for cellular growth and survival: application to proliferation and cytotoxicity assays. J. Immunol. Methods 65, 55–63 (1983).