Research Interests. Transport and delivery of macromolecular drugs. Antisense technology. Combinatorial peptide libraries for gene regulation.

• Fisher, M., Abramov, M., Van Aerschot, A., Xu, D., Juliano, R.L., Herdewijn, P. Inhibition of MDR1 expression with altritol-modified siRNAs. Nuc Acid Res. 2007, Jan 30 [epub].
• Juliano RL. Intracellular delivery of oligonucleotide conjugates and dendrimer complexes. Ann NY Acad Sci. 2006 1082:18-26.
• Kang H, DeLong R, Fisher MH, Juliano RL. Tat-conjugated PAMAM dendrimers as delivery agents for antisense and siRNA oligonucleotides. Pharm Res. 2005 22(12): 2099-106.
• Juliano RL. Peptide-oligonucleotide conjugates for the delivery of antisense and siRNA. Curr Opin Mol Ther. 2005 7(2):132-75.
• Xu D, McCarty D, Fernandes A, Fisher M, Samulski RJ, Juliano RL. Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector. Mol Ther. 2005 11(4):523-30.
• Falke D, Fisher MH, Juliano RL. Selective transciption of p53 target genes y zinc finger-p53 DNA binding domain chimeras. Biochim Biophys Acta. 2004 1681(1):15-27.
• Kang H, Fisher MH, Xu D, Miyamoto YJ, Marchand A, Van Aerschot A, Herdewijn P, Juliano RL. Inhibition of MDR1 gene expression by chimeric HNA antisense oligonucleotides. Nucleic Acids Res. 2004 32(14):4411-9.
• Xu D, Kang H, Fisher M, Juliano RL. Strategies for inhibitino of MDR1 gene expression. Mol Pharmacol. 2004 66(2):268-75.

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Research Interests: RNA splicing; therapeutic regulation of splicing with oligonucleotides.

• Roberts J, Palma E, Sazani P, Orum H, Cho M, Kole R. Efficient and persistent splice switchining by systemically delivered LNA oligonucleotides in mice. Mol Ther. 2006 14(4)471-5.
• Williams T, Kole R. Analysis of prostate -specific membrane antigen splice variants in LNCap cells. Oligonucleotides. 2006 16(2):186-95.
• Kole R, Vacek M, Williams T. Modification of alternative splicing by antisense therapeutics. Oligonucleotides. 2004 14(1): 65-74.
• Sazani P, Astriab-Fischer A, Kole R. Effects of base modifications on antisense properties of 2'-O-methozyenthyl and PNA oligonucleotides. Antisense Nucleic Acid Drug Dev. 2003 13(3):119-28.
• Sazani P, Kole R. Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. J Clin Invest. 2003 112(4):481-6.

Research Interests: molecular biology of AAV; gene therapy with AAV.

• Choi VW, McCarty DM, Samulski RJ. Host cell DNA repair pathways in adeno-associated viral genome processing. J Virol 2006 80(21):10346-56.
• Wu Z, Asokan A, Grieger JC, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Single amino acid change scan influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol. 2006 80(22):11393-7.
• Wu Z, Miller E, Agbandje-McKenna M, Samulski RJ. Alpha2,3 and alpha 2,6 N-linked aialic acids facilitate efficient binding and transuctino by adeno-associated virus types 1 and 6. J Virol. 2006 80(18):9093-103.
• Asokan A, Hamra JB, Govindasamy L, Agbandije-McKenna M, Samulski RJ. Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry. J Virol. 2006 80(18):8961-9.
• Wu Z, Asolan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther. 2006 14(3):316-27.
• Grieger JC, Snowdy S, Samulski RJ. Separate basic region motifs within the adeno-associated virus capsid proteins are essential for infectivity and assembly. J Virol 2006 80(11):5199-210.
• Grieger JC, Samulski RJ. Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. Adv Biochem Eng Biotechnol. 2005 99:119-45.
• Lehmann TG, Luedde T, Schwabe RE, Bunzendahl H, Samulski RJ, Lemasters JJ, Brenner DA. Minimizing oxidative stress by gene delivery of superoxide dismutase accelerates regeneration after transplantation of reduced-size livers in the rat. LIver Transpl. 2006 12(4):550-9.
• McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, Shera D, Lioutermann L, Freely M, Freese A, Leone P. Immune responses to AAV in a phase I study for Canavan disease. J Gene Med. 2006 8(5):577-88.
• Borras T, Xue W, Choi VW, Bartlett JS, Li G, Samulski RJ, Chisolm SS. Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells. J Gene Med 2006 8(5):589-602.
• Asokan A, Samulski RJ. AAV does the shuffle. Nat Biotechnol. 2006 24(2):158-60.
• Chen S, Kapturczak M, Loiler S, Zolotukhin S, Glushakova O, Madsen K, Samulski RJ, Hauswirth W, Campbell-Thompson M, Berns K, Flotte T, Atkinson M, Tisher C, Agarwal A. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Human Gene Therapy, 16(2):235-47, 2005.
• Xu D, McCarty D, Fernandes A, Fisher M, Samulski RJ, Juliano RL. Delivery of MDR1 small interfering RNA by self-complimentary recombinant adeno-associated virus vector. Molecular Therapy, 11(4):523-30, 2005.
• Li C, Samulski RJ. Serotype-specific replicating AAV helper constructs increase recombinant AAV type 2 vector production. Virology, 335:10-21, 2005.
• Choi VW, McCarty DM, Samulski RJ. AAV hybrid serotypes: improved vectors for gene delivery. Current Gene Therapy, 5(3):299-310, 2005.
• Asokan A, Samulski RJ. From crystal structure to clinic: Highlights of the Tenth International Parvovirus Workshop. Molecular Therapy, 11(5):656-60, 2005.
• Grieger J, Samulski RJ. Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and post-entry steps. Journal of Virology, 79(15):9933-44, 2005.

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Research Interests: RNA repair, RNA ligands that block protein function and therapeutic applications of RNAi.

• Mi J, Zhang X, Rabbani ZN, Liu Y, Su Z, Vujaskovic Z, Kontos CD, Sullenger BA, Clary BM. H1 RNA polymerase III promoter-driven expression of an RNA aptamer leads to high-level inhibition of intracellular protein activity. Nucleic Acids Res. 2006 34(12):3577-84.
• McNamara JO 2nd, Andrechek ER, Wang Y, Viles KD, Rempel RE, Gilboa E, Sullenger BA, Gianjgrande PH. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat Biotechnol. 2006 24(8):1005-15.
• Nimjee SM, Keys JR, Pitoc GA, Quick G, Rusconi CP, Sullenger BA. A novel antidote-controlled anticoagulant reduces thrombin generation and inflammation and improves cardiac functino in cardiopulmonary bypass surgery. Mol Ther. 2006 14(3):408-15.
• Mi J, Zhang X, Giangrande PH, McNamara JO 2nd, Nimjee SM, Sarraf-Yazdi S, Sullenger BA, Clary BM. Targeted inhibition of aophavbeta3 integrin with an RNA aptamer impairs endothelial cell growth and survival. Biochem Biophys Res Commun. 2005 338(2):956-63.
• Becker RC, Rusconi C, Sullenger B. Nucleic acid aptamers in therapeutic anticoagulation. Technology, devlopment and clinical application. Thromb Haemost. 2005 11(5):687-94.
• Jones JP 3rd, Kierlin MN, Coon RG, Perutka J, Lambowitz AM, Sullenger BA. Retargeting mobile group II introns to repair mutant genes. Mol Ther. 2005 11(5):687-94.
• Shin KS, Sullenger BA, Lee SW. Ribozyme-mediated inductino of apoptosis in human cancer cells by targeted repair of mutant p53 RNA. Mol Ther. 2004 10(2):365-72.
• Jeter ML, Ly LV, Fortenberry YM, Whinna HC, White RR, Rusconi CP, Sullenger BA, Church FC. RNA aptamer to thrombin binds anion-binding exosite-2 and alters protease inhibitino by heparin-binding serpins. FEBS Lett. 2004 568(1-3):10-14.
• Layzer J, McCafferey A, Tanner A, Kay M, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. RNA 2004 10(50):766-71.
• Rusconi CP, Roberts D, Pitoc GA, Nimjee S, White RR, Quick G, Scardino E, Fay W, Sullenger BA. Antidote-mediated control of anticoagulant aptamer activity in vivo. Nature Biotechnology 2004 22:1423-1428.

Research Interests: Delivery of antisense oligonucleotides and proteins.

• Asokan A, and Cho MJ. Cytosolic Delivery of Macromolecules: 4. Head Group-Dependent Membrane Permeabilization by pH-Sensitive Detergents. J Controlled Release. 2005 106:146-153.
• Palma, E., Klapper, D.G., and Cho, M.J.: Antibodies as Drug Carriers III: Design of Oligonucleotides with Enhanced Binding Affinity for Immunoglobulin G. Pharm. Res. 22: 122-127, 2005.
• Asokan A, Cho MJ. Cytosolic Delivery of Macromolecules: III. Synthesis and Characterization of Acid-Sensitive Bis-Detergents. Bioconj Chem. 2004 15:1166-1173.
• Riché E, Erickson BW, Cho MJ. Novel Long-Circulating Liposomes Containing Peptide Library-Lipid Conjugates: Synthesis and in Vivo Behavior. J. Drug Targeting 2004 12:355-361.
• Asokan A. Cho MJ. Cytosolic Delivery of Macromolecules: II. Mechanistic Studies with pH-Sensitive Morpholine Lipids. Biochim Biophys Acta 2003 1611:151-160.

Research Interests: Polymer Synthesis, Liquid and Supercritical CO2 Processing, Green Chemistry.

• Olson DA, Gratton SEA, DeSimone JM, Sheares VV. Amorphous Linear Aliphatic Polyesters for the Facile Preparation of Tunable Rapidly Degrading Elastomeric Devices and Delivery Vectors. J Am Chem Soc. 2006 128:13625-33.
• Zhang L, Ahou Z, Cheng B, DeSimone JM, Samulski ET. Superhydrophobic Behavior of a Perfluoropolyether Lotus-Leaf-Like Topography. Langmuir 2006 22:8576-80.
• Zhou Z, Dominey RN, Rolland JP, Maynor BW, Pandya AA, DeSimone JM. Molded, High Surface Area Polymer Electrolyte Membranes from Cured Liquid Precursors. J Am Chem Soc. 2006 128:12963-72.
• Euliss LE, DuPont JA, Gratton S, DeSimone J. Imparting size, shape and composition control of materials for nanomedicine. Chem Soc Rev. 2006 35(11):1095-104.
• Yarbrough JC, Rolland JP, DeSimone JM, Callow ME, Finlay JA, Callow JA. Contact Angle Analysis, Surface dynamics, and Biofouling Characteristics of Cross-linkable, Random Perfluoropolyether-based Grant Ter-polymers. Macromolecules. 2006 39:2521-28.
• Boggiano MK, Vellenga D, Carbonell RG, Sheares-Ashby V, DeSimone JM. Alicyclic Photoresists for CO2-based Next-Generation Microlithography: A Tribute to James E. McGrath. Polymer. 2006 47:4012-17.
• Rolland JP, Maynor BW, Euliss LE, Exner AE, Denison GM, DeSimone JM. Direct fabrication and harvesting of monodisperse, shape-specific nanobiomaterials. J Am Chem Soc. 2005 127(28):10096-100.
• Young JL, DeSimone JM. Advangtages of Supercritical Carbon Dioxide for Composite Particle Synthesis Using Water-Soluble or Water-Reactive Monomers. Macromolecules. 2005 38:4542-44.

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Research Interests: Oligonucleotide chemistry and antisense and RNAi gene silencing.

• Hall AH, Wan J, Spesock A, Sergueeva Z, Shaw BR, Alexander KA. High potency silencing by single-stranded boranophosphate siRNA. Nucleic Acids Res. 2006 34(9):108-19.
• Li P, Xu Z, Liu H, Wennefors CK, Dobrikov MI, Ludwig J, Shaw BR. Synthesis of alpha-P-modified nucleosides diphosphates with ethylenediamine. J Am Chem Sioc 2005 127(48):16782-3.
• Khan SI, Dobrikov MI, Shaw BR. Synthesis of 5-ethynyl-2'-deoxyuridine-5'-boranomono phosphate as a potential thymidylate synthase inhibator. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):1047-9.
• Dobrikov MI, Shaw BR. Affinity of (alpha-P-borano)-NTP analogs to rabbit muscle pyruvate kinase. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):983-7.
• Wang JX, Sergueev DS, Shaw BR. The effect of a single boranophosphate substitution with a defined configuration on the thermal stability and conformation of a DNA duplex. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):9951-5.
• Wang JX, Shaw BR. Synthesis of 5-(1-propynyl)-2'-deoxyuridine 5'-(alpha-P-borano) triphosphate and kineetic characterization as a substrate for mmlv reverse transcriptase. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):947-50.
• Wan J, Shaw BR. Incorporation of ribonucleoside 5'-(alpha-P-borano) triphosphates into a 20-mer RNA by T7 RNA polymerase. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):943-6.
• Li P, Shaw Br. Model synthesis of nucleoside bornaophosphoramidate with amino acid for prodrug purpose. Nucleosides Nucleosides Nucleic Acids. 2005 24(5-7):675-8.
• Summers JS, Base, K, Boukhalfa H, Payne JE, Shaw BR, Crumbliss AL. Use of phosphorus ligand NMR probes to investigate electronic and second-sphere solvent effects in ligand substitution reactinos at manganese (II) and manganese (III). Inorg Chem. 2005 44(10):3405-11.
• Li P, Shaw BR. Synthesis of nucleoside boranophosphoramidate prodrugs conjugated with amino acids. J Org Chem. 2005 70(6):2171-83.
• Shaw BR, Dobrikov M, Sergueev D, Sergueeva Z, Wan J, Wang X, He K, Porter K, Li P, Lin J-L, Reading, Writing, and Modulating Genetic Information with Boranophosphate Mimics of Nucleotides, DNA, and RNA, in Therapeutic Oligonucleotides: Antisense, RNAi, Triple Helix, DNA Decoys, and DNA Chips, ed Y.S. Cho-Chung, A. M. Gewirtz, and C. A. Stein. Annals of the New York Academy of Sciences. 2004 1002:12-29. (ANNALS ONLINE, www.annalsnyas.org)
• Li P, Shaw BR. Convenient Synthesis of Nucleoside Borane Diphosphate Analogues: Deoxy- and Ribonucleoside 5'-Pa-Boranodiphosphates.. J. Organic Chem. 2004 69:7051-7057.
• Hall A, Wan J, Shaughnessy E, Shaw BR, Alexander KA. RNA Interference Using Boranophosphate siRNAs: Structure Activity Relationships. Nucleic Acids Res. 2004 32:5991-6000.
• Li P, Dobrikov M, Liu H, Shaw BR. Synthesis of acyclothymidine triphosphate and alpha-P-boranotriphosphate and their substrate properties with retroviral reverse transcriptase. Org Lett. 2003 5(14):2401-3.