Where science translates to action

Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes.
Fenton OS, Kauffman KJ, Kaczmarek JC, et al. Adv Mater. 2017 Sep;29(33).doi: 10.1002/adma.201606944. Epub 2017 Jul 6.

Polymer-lipid nanoparticles for systemic delivery of mRNA to the lungs.
Kaczmarek JC, Patel AK, Kauffman KJ, et al. Angew Chem Int Ed Engl. 2016 Oct 24;55(44):13808-13812. doi: 10.1002/anie.201608450. Epub 2016 Sep 30.

Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo.
Kauffman KJ, Mir FF, Jhunjhunwala S, et al. Biomaterials. 2016 Dec;109:78-87. doi: 10.1016/j.biomaterials.2016.09.006. Epub 2016 Sep 25.

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system.
DeRosa F, Guild B, Karve S, et al. Gene Ther. 2016 Oct;23(10):699-707. doi: 10.1038/gt.2016.46. Epub 2016 Jun 30.

Bioinspired alkenyl amino alcohol ionizable lipid materials for highly potent in vivo mRNA delivery.
Fenton OS, Kauffman KJ, McClellan RL, et al. Adv Mater. 2016 Apr 20;28(15):2939-2943. doi: 10.1002/adma.201505822. Epub 2016 Feb 18.

Poly(glycoamidoamine) brushes formulated nanomaterials for systemic siRNA and mRNA delivery in vivo.
Dong Y, Dorkin JR, Wang W, et al. Nano Lett. 2016 Feb 10;16(2):842-848. doi: 10.1021/acs.nanolett.5b02428. Epub 2016 Jan 13.

Optimization of lipid nanoparticle formulations for mRNA delivery in vivo with fractional factorial and definitive screening designs.
Kauffman KJ, Dorkin JR, Yang JH, et al. Nano Lett. 2015 Nov 11;15(11):7300-7306. doi: 10.1021/acs.nanolett.5b02497. Epub 2015 Oct 20.

Oligonucleotide-based Approach Publications

The Therapeutic Targeting of Long Noncoding RNA.
Woo C.J. RNA Therapeutics. Topics in Medicinal Chemistry, vol 27. 2017. doi: 10.1007/7355_2017_25. Epub. 2017 July 25.

Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy.
Woo CJ, Maier VK, Davey R, et al. Proc Natl Acad Sci USA. 2017 Feb 21;114(8):E1509-E1518. doi: 10.1073/pnas.1616521114. Epub 2017 Feb 13.

Enhancing antisense efficacy with multimers and multi-targeting oligonucleotides (MTOs) using cleavable linkers.
Subramanian RR, Wysk MA, Ogilvie KM, et al. Nucleic Acids Res. 2015 Oct 30;43(19):9123-9132. doi: 10.1093/nar/gkv992. Epub 2015 Oct 7.

Genome-wide identification of polycomb-associated RNAs by RIP-seq.
Zhao J, Ohsumi TK, Kung JT, et al. Mol Cell. 2010 Dec 22;40(6):939-953. doi: 10.1016/j.molcel.2010.12.011.

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