Medicinal Chemistry/Formulations and Manufacturing

Human Dicer Binds Short Single-strand and Double-strand RNA with High Affinity and Interacts with Different Regions of the Nucleic Acids.

Lima WF, Murray H, Nichols JG, Wu H, Sun H, Prakash TP, Berdeja AR, Gaus HJ, Crooke ST.

J Biol Chem. 2009 Jan 23;284(4):2535-48

Short Antisense Oligonucleotides with Novel 2'-4' Conformationaly Restricted Nucleoside Analogues Show Improved Potency Without Increased Toxicity in Animals.

Seth PP, Siwkowski A, Allerson CR, Vasquez G, Lee S, Prakash TP, Wancewicz EV, Witchell D, Swayze EE.

Journal of Medicinal Chemistry. 2008 December [Epub ahead of print].

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Hua Y, Vickers TA, Okunola HL, Bennett CF, Krainer AR.

AJHG. 2008 April 11;82(4):834-848.

Oral delivery of antisense oligonucleotides in man.

Tillman LG, Geary RS, Hardee GE.

J Pharm Sci. 2008 Jan;97(1):225-236.

A Novel Universal Linker for Efficient Synthesis of Phosphorothioate Oligonucleotides.

Wang Z, Olsen P, Ravikumar VT.

Nucleosides Nucleotides Nucleic Acids. 2007;26(3):259-69.

Synthesis of antisense oligonucleotides with minimum depurination.

Krotz, A. H., Cole, D.L. , Ravikumar, V. T.

Nucleosides Nucleotides Nucleic Acids. 22(2):129-34, 2003.

bis-Cholesteryl-conjugated phosphorothioate oligodeoxynucleotides are highly selectively taken up by the liver

Bijsterbosch, M.K., Manoharan, M., Dorland, R., Van Veghel, R., Biessen, E.A., Van Berkel, T.J.

J Pharmacol Exp Ther. 302(2):619-26, 2002.

Nuclease resistance of oligonucleotides containing the tricyclic cytosine analogues phenoxazine and 9-(2-aminoethoxy)-phenoxazine (""G-clamp"") and origins of their nuclease resistance properties.

Maier, M.A., Leeds, J.M., Balow, G., Springer, R.H., Bharadwaj, R., and Manoharan, M.

Biochemistry. 2002 Jan 29;41(4):1323-7.

Peptide Nucleic acids are potent modulators of endogenous pre-mrna splicing of the murine interleukin-5 receptor-alpha chain.

Karras J.G., Maier, M.A., Lu, T., Watt, A., Manoharan, M.

Biochemistry. 2001 Jul 3;40(26):7853-9.

Medicinal chemistry of antisense oligonucleotides

Cook, P. D.

. Antisense drug technology: Principles, strategies, and applications (ed. Crooke, S. T.), pp. 29-56. Marcel Dekker, New York, 2001.

Oligonucleotide conjugates in antisense technology.

Manoharan, M.

Antisense drug technology: Principles, strategies, and applications (ed. Crooke, S. T.), pp. 391-470. Marcel Dekker, New York, 2001.

Nuclear antisense effects of neutral anionic and cationic oligonucleotide analogues.

Sazani, P., Kang, S.H., Maier, M.A., Wei, C., DillmAn, J., Manoharan, M., Kole, R.,

Nucleic Acid Res. (29) 19: 3965-3974, 2001.

Multiple oligonucleotides synthesis in tandem on solid-phase supports for small and large scale synthesis.

Pon, R.T., Yu, S., Sanghvi, Y.S.

Nucleosides Nucleotides Nucleic Acids. 20(4-7):985-989, 2001.

Applications of green chemistry in the manufacture of oligonucleotide drugs.

Sanghvi, Y.S., Ravikumar, VT., Scozzari, A.N., Cole, D.L.

Pure App Chem. 73(1):175-180, 2001.

Intercellular adhesion molecule-1 suppression in skin by topical delivery of anti-sense oligonucleotides.

Mehta, R.C., Stecker, K.K., Cooper, S.R., Templin, M.V., Tsai, Y.J., Condon, T.P., Bennett, C.F., Hardee, G.E.

J Invest Dermatol.115(5):805-812, 2000.

Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice.

Templin, M. V., Levin, A. A., Graham, M. J., Aberg, P. M., Axelsson, B. I., Butler, M., Geary, R. S., and Bennett, C. F.

Antisense Nucleic Acid Drug Dev. 10(5):359-368, 2000.