Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers

Methods in Molecular Biology

Volumn 867, Issue , 2012, Pages 407-414

  Moulton, Hong M ^a  

^a OREGON STATE UNIVERSITY   (United States)

Author keywords

Antisense oligo; Cell penetrating peptide; Conjugation; CPP; Duchenne muscular dystrophy; Exon skipping; Heart; Morpholino; Muscle; Systemic delivery

Indexed keywords

CELL PENETRATING PEPTIDE; DYSTROPHIN; MORPHOLINO OLIGONUCLEOTIDE;

AMINO ACID SEQUENCE; ANIMAL; ARTICLE; CHEMISTRY; DRUG DELIVERY SYSTEM; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENETICS; HEART MUSCLE; METABOLISM; METHODOLOGY; MOLECULAR GENETICS; MOUSE; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

AMINO ACID SEQUENCE; ANIMALS; CELL-PENETRATING PEPTIDES; DRUG DELIVERY SYSTEMS; DYSTROPHIN; EXONS; MICE; MICE, INBRED MDX; MOLECULAR SEQUENCE DATA; MORPHOLINOS; MUSCULAR DYSTROPHY, DUCHENNE; MYOCARDIUM;

ANIMALIA; MUS;

EID: 84859816424     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-61779-767-5_26     Document Type: Article

References (16)

1. Summerton J, Weller D (1997) Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug Dev 7:187-195 (Pubitemid 27323077)
2. Hudziak RM, Barofsky E, Barofsky DF, Weller DL, Huang SB, Weller DD (1996) Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation. Antisense Nucleic Acid Drug Dev 6:267-272 (Pubitemid 27045903)
3. Youngblood DS, Hatlevig SA, Hassinger JN, Iversen PL, Moulton HM (2007) Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells. Bioconjug Chem 18:50-60 (Pubitemid 46145994)
4. Bill BR, Petzold AM, Clark KJ, Schimmenti LA, Ekker SC (2009) A primer for morpholino use in zebrafish. Zebrafish 6:69-77
5. Yin H, Moulton HM, Seow Y, Boyd C, Boutilier J, Iverson P et al (2008) Cellpenetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 17:3909-3918 (Pubitemid 352762853)
6. Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD et al (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12: 175-177 (Pubitemid 43214742)
7. Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S et al (2009) Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs. Ann Neurol 65:667-676
8. Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C et al (2009) Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebocontrolled, dose-escalation, proof-of-concept study. Lancet Neurol 8:918-928
9. Moulton HM, Hase MC, Smith KM, Iversen PL (2003) HIV Tat peptide enhances cellular delivery of antisense morpholino oligomers. Antisense Nucleic Acid Drug Dev 13:31-43
10. Moulton HM, Moulton JD (2010) Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy. Biochim Biophys Acta 1798: 2296-303
11. Moulton HM, Nelson MH, Hatlevig SA, Reddy MT, Iversen PL (2004) Cellular uptake of antisense morpholino oligomers conjugated to arginine-rich peptides. Bioconjug Chem 15:290-299 (Pubitemid 38365188)
12. Wu B, Moulton HM, Iversen PL, Jiang J, Li J, Spurney CF et al (2008) Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci USA 105:14814-14819
13. Yin H, Moulton HM, Betts C, Seow Y, Boutilier J, Iverson PL et al (2009) A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum Mol Genet 18:4405-4414
14. Goyenvalle A, Babbs A, Powell D, Kole R, Fletcher S, Wilton SD et al (2009) Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. Mol Ther 18:198-205
15. Sazani P, Blouch R, Weller D, Leow S, Kole R, Shrewsburry S (2009) AVI 5038: initial efficacy and safety evaluation in cynomolgus monkeys. Treat-NMD/NIH conference 2009. Brussels, Belgium
16. Abes S, Moulton HM, Clair P, Prevot P, Youngblood DS, Wu RP et al (2006) Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents. J Control Release 116: 304-313 (Pubitemid 44827406)