Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophyHong M Moulton
AVI BioPharma, Corvallis, Oregon 97333, USA
Ann N Y Acad Sci 1175:55-60. 2009
..No toxicity or immunogenicity was detected. Our studies demonstrated that muscle functions can be restored with a low dose of PPMO, making it a promising therapeutic for DMD...
- Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate
Adams Amantana
AVI BioPharma, Inc, Corvallis, Oregon, USA
Bioconjug Chem 18:1325-31. 2007
..In this study, we investigated the pharmacokinetics, tissue distribution, stability, and safety profile of an anti-c-myc PMO conjugated to the CPP, (RXR)4 (X = 6-aminohexanoic acid) in rats...
- Cell-penetrating peptides as transporters for morpholino oligomers: effects of amino acid composition on intracellular delivery and cytotoxicity
Rebecca P Wu
AVI BioPharma, Inc, Corvallis, OR 97333, USA
Nucleic Acids Res 35:5182-91. 2007
..Toxicity of X/B-containing conjugates was affected by the number of Xs, treatment time and concentration. More active, stable and less toxic CPPs can be designed by optimizing the position and number of R, D-R, X and B residues...
- Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells
Derek S Youngblood
AVI BioPharma, Incorporated, 4575 SW Research Way, Corvallis, Oregon 97333, USA
Bioconjug Chem 18:50-60. 2007
..Last, the endosomal/lysosomal trapping limits the effectiveness of a CPP-PMO conjugate, and the stability of the CPP is one of the factors affecting the ability of the conjugate to escape the endosomes/lysosomes...
- Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy
Hong M Moulton
College of Veterinary Medicine, Oregon State University, Corvallis, OR 97331, USA
Biochim Biophys Acta 1798:2296-303. 2010
..The toxicity of PPMOs will require caution when moving into the clinic. The first PPMO-based DMD drug is currently in preclinical development for DMD patients who can benefit from skipping exon 50...
- Arginine-rich peptide conjugation to morpholino oligomers: effects on antisense activity and specificity
Michelle H Nelson
AVI BioPharma, Inc, Corvallis, Oregon 97333, USA
Bioconjug Chem 16:959-66. 2005
..Additionally, it is shown that the enzymatic stability of an L- or unnatural peptide used for PMO conjugation affects the antisense properties of the resulting compound...
