Detalhe da pesquisa
1.
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 114(16): 4213-4218, 2017 04 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-28373570
2.
AVI-7288 for Marburg Virus in Nonhuman Primates and Humans.
N Engl J Med
; 373(4): 339-48, 2015 Jul 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-26200980
3.
Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies.
Antimicrob Agents Chemother
; 58(11): 6639-47, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25155593
4.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Ann Neurol
; 74(5): 637-47, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23907995
5.
Oligonucleotide therapeutics in cancer.
Cancer Treat Res
; 158: 213-33, 2013.
Artigo
em Inglês
| MEDLINE | ID: mdl-24222360
6.
Chemical and mechanistic toxicology evaluation of exon skipping phosphorodiamidate morpholino oligomers in mdx mice.
Int J Toxicol
; 30(3): 322-33, 2011 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-21540335
7.
Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy.
Int J Toxicol
; 30(3): 313-21, 2011 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-21540336
8.
Safety pharmacology and genotoxicity evaluation of AVI-4658.
Int J Toxicol
; 29(2): 143-56, 2010.
Artigo
em Inglês
| MEDLINE | ID: mdl-20110565
9.
Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells.
Int J Cancer
; 124(4): 772-7, 2009 Feb 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-19035464
10.
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
Mol Ther
; 16(9): 1624-9, 2008 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-18545222
11.
An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.
Mol Ther
; 16(7): 1316-22, 2008 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-18461057
12.
An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models.
Mol Ther
; 16(7): 1316-1322, 2008 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28178484
13.
Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.
J Clin Invest
; 112(4): 481-6, 2003 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-12925686
14.
Systemically delivered antisense oligomers upregulate gene expression in mouse tissues.
Nat Biotechnol
; 20(12): 1228-33, 2002 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-12426578
15.
Newborn screening for Duchenne muscular dystrophy in China: follow-up diagnosis and subsequent treatment.
World J Pediatr
; 13(3): 197-201, 2017 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-28466241
16.
Temperature-dependent splicing of beta-globin pre-mRNA.
Nucleic Acids Res
; 30(21): 4592-8, 2002 Nov 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-12409448
17.
Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates.
J Neuromuscul Dis
; 3(3): 381-393, 2016 08 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27854228
18.
Short-term and long-term modulation of gene expression by antisense therapeutics.
Curr Opin Biotechnol
; 13(5): 468-72, 2002 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-12459339
19.
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.
Cardiovasc Res
; 85(3): 444-53, 2010 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-19815563
20.
Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.
Ann N Y Acad Sci
; 1175: 55-60, 2009 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-19796077