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Items: 32

1.

Quantitative determination of AVI-7100 (Radavirsen), a phosphorodiamidate morpholino oligomer (PMOplus® ), in human plasma using LC-MS/MS.

Meng M, Zhang J, Liu A, Reuschel S, Sazani P, Wong M.

Bioanalysis. 2017 May;9(10):827-839. doi: 10.4155/bio-2016-0289. Epub 2017 May 18.

PMID:
28520455
2.

Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment.

Ke Q, Zhao ZY, Griggs R, Wiley V, Connolly A, Kwon J, Qi M, Sheehan D, Ciafaloni E, Howell RR, Furu P, Sazani P, Narayana A, Gatheridge M.

World J Pediatr. 2017 Jun;13(3):197-201. doi: 10.1007/s12519-017-0036-3. Epub 2017 May 17. Review.

PMID:
28466241
3.

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.

Echigoya Y, Nakamura A, Nagata T, Urasawa N, Lim KRQ, Trieu N, Panesar D, Kuraoka M, Moulton HM, Saito T, Aoki Y, Iversen P, Sazani P, Kole R, Maruyama R, Partridge T, Takeda S, Yokota T.

Proc Natl Acad Sci U S A. 2017 Apr 18;114(16):4213-4218. doi: 10.1073/pnas.1613203114. Epub 2017 Apr 3.

4.

Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates.

Carver MP, Charleston JS, Shanks C, Zhang J, Mense M, Sharma AK, Kaur H, Sazani P.

J Neuromuscul Dis. 2016 Aug 30;3(3):381-393.

PMID:
27854228
5.

AVI-7288 for Marburg Virus in Nonhuman Primates and Humans.

Heald AE, Charleston JS, Iversen PL, Warren TK, Saoud JB, Al-Ibrahim M, Wells J, Warfield KL, Swenson DL, Welch LS, Sazani P, Wong M, Berry D, Kaye EM, Bavari S.

N Engl J Med. 2015 Jul 23;373(4):339-48. doi: 10.1056/NEJMoa1410345.

6.

A single phosphorodiamidate morpholino oligomer targeting VP24 protects rhesus monkeys against lethal Ebola virus infection.

Warren TK, Whitehouse CA, Wells J, Welch L, Heald AE, Charleston JS, Sazani P, Reid SP, Iversen PL, Bavari S.

mBio. 2015 Feb 10;6(1). pii: e02344-14. doi: 10.1128/mBio.02344-14.

7.

Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies.

Heald AE, Iversen PL, Saoud JB, Sazani P, Charleston JS, Axtelle T, Wong M, Smith WB, Vutikullird A, Kaye E.

Antimicrob Agents Chemother. 2014 Nov;58(11):6639-47. doi: 10.1128/AAC.03442-14. Epub 2014 Aug 25.

8.

Oligonucleotide therapeutics in cancer.

Wan J, Bauman JA, Graziewicz MA, Sazani P, Kole R.

Cancer Treat Res. 2013;158:213-33. doi: 10.1007/978-3-642-31659-3_9.

PMID:
24222360
9.

Eteplirsen for the treatment of Duchenne muscular dystrophy.

Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group.

Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10.

10.

Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy.

Sazani P, Ness KP, Weller DL, Poage DW, Palyada K, Shrewsbury SB.

Int J Toxicol. 2011 May;30(3):313-21. doi: 10.1177/1091581811403505. Epub 2011 May 3.

PMID:
21540336
11.

Chemical and mechanistic toxicology evaluation of exon skipping phosphorodiamidate morpholino oligomers in mdx mice.

Sazani P, Ness KP, Weller DL, Poage D, Nelson K, Shrewsbury AS.

Int J Toxicol. 2011 May;30(3):322-33. doi: 10.1177/1091581811403504. Epub 2011 May 3.

PMID:
21540335
12.

Safety pharmacology and genotoxicity evaluation of AVI-4658.

Sazani P, Weller DL, Shrewsbury SB.

Int J Toxicol. 2010 Mar-Apr;29(2):143-56. doi: 10.1177/1091581809359206. Epub 2010 Jan 28.

PMID:
20110565
13.

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

Jearawiriyapaisarn N, Moulton HM, Sazani P, Kole R, Willis MS.

Cardiovasc Res. 2010 Feb 1;85(3):444-53. doi: 10.1093/cvr/cvp335. Epub 2009 Oct 8.

14.

Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.

Moulton HM, Wu B, Jearawiriyapaisarn N, Sazani P, Lu QL, Kole R.

Ann N Y Acad Sci. 2009 Sep;1175:55-60. doi: 10.1111/j.1749-6632.2009.04976.x.

PMID:
19796077
15.

Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells.

Wan J, Sazani P, Kole R.

Int J Cancer. 2009 Feb 15;124(4):772-7. doi: 10.1002/ijc.24052.

16.

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models.

Graziewicz MA, Tarrant TK, Buckley B, Roberts J, Fulton L, Hansen H, Ørum H, Kole R, Sazani P.

Mol Ther. 2008 Jul;16(7):1316-1322. doi: 10.1038/mt.2008.85. Epub 2016 Dec 8.

PMID:
28178484
17.

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S, Iversen PL, Kole R.

Mol Ther. 2008 Sep;16(9):1624-9. doi: 10.1038/mt.2008.120. Epub 2008 Jun 10.

18.

An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.

Graziewicz MA, Tarrant TK, Buckley B, Roberts J, Fulton L, Hansen H, Ørum H, Kole R, Sazani P.

Mol Ther. 2008 Jul;16(7):1316-22. doi: 10.1038/mt.2008.85. Epub 2008 May 6.

19.

Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice.

Roberts J, Palma E, Sazani P, Ørum H, Cho M, Kole R.

Mol Ther. 2006 Oct;14(4):471-5. Epub 2006 Jul 18.

20.

A small aptamer with strong and specific recognition of the triphosphate of ATP.

Sazani PL, Larralde R, Szostak JW.

J Am Chem Soc. 2004 Jul 14;126(27):8370-1.

21.

Effects of base modifications on antisense properties of 2'-O-methoxyethyl and PNA oligonucleotides.

Sazani P, Astriab-Fischer A, Kole R.

Antisense Nucleic Acid Drug Dev. 2003;13(3):119-28.

PMID:
12954112
22.
23.

Antisense-mediated redirection of mRNA splicing.

Vacek M, Sazani P, Kole R.

Cell Mol Life Sci. 2003 May;60(5):825-33. Review.

PMID:
12827272
24.

Modulation of alternative splicing by antisense oligonucleotides.

Sazani P, Kole R.

Prog Mol Subcell Biol. 2003;31:217-39. Review. No abstract available.

PMID:
12494768
25.

Polymeric nanogels produced via inverse microemulsion polymerization as potential gene and antisense delivery agents.

McAllister K, Sazani P, Adam M, Cho MJ, Rubinstein M, Samulski RJ, DeSimone JM.

J Am Chem Soc. 2002 Dec 25;124(51):15198-207.

PMID:
12487595
26.

Short-term and long-term modulation of gene expression by antisense therapeutics.

Sazani P, Vacek MM, Kole R.

Curr Opin Biotechnol. 2002 Oct;13(5):468-72. Review.

PMID:
12459339
27.

Systemically delivered antisense oligomers upregulate gene expression in mouse tissues.

Sazani P, Gemignani F, Kang SH, Maier MA, Manoharan M, Persmark M, Bortner D, Kole R.

Nat Biotechnol. 2002 Dec;20(12):1228-33. Epub 2002 Nov 11.

PMID:
12426578
28.

Temperature-dependent splicing of beta-globin pre-mRNA.

Gemignani F, Sazani P, Morcos P, Kole R.

Nucleic Acids Res. 2002 Nov 1;30(21):4592-8.

29.

Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases.

Mercatante DR, Sazani P, Kole R.

Curr Cancer Drug Targets. 2001 Nov;1(3):211-30. Review.

PMID:
12188880
30.

Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs.

Sazani P, Kang SH, Maier MA, Wei C, Dillman J, Summerton J, Manoharan M, Kole R.

Nucleic Acids Res. 2001 Oct 1;29(19):3965-74.

31.

Antisense effects in the cell nucleus: modification of splicing.

Kole R, Sazani P.

Curr Opin Mol Ther. 2001 Jun;3(3):229-34. Review.

PMID:
11497345
32.

PAMAM dendrimers as delivery agents for antisense oligonucleotides.

Yoo H, Sazani P, Juliano RL.

Pharm Res. 1999 Dec;16(12):1799-804.

PMID:
10644065

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