Optimizing tissue-specific antisense oligonucleotide-peptide conjugates

Methods in Molecular Biology

Volumn 867, Issue , 2012, Pages 415-435

  Betts, Corinne A ^a   Hammond, Suzan M ^a   Yin, Hai Fang ^a,b   Wood, Matthew J A ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b TIANJIN MEDICAL UNIVERSITY   (China)

Author keywords

Antisense oligonucleotide; B MSP PMO; Cell penetrating peptide; Immunohistochemical staining; Intramuscular injection; Intravenous injection; RT PCR; Western blot

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CELL PENETRATING PEPTIDE; DYSTROPHIN;

ANIMAL; ARTICLE; CELL CULTURE; CELL LINE; CHEMISTRY; DUCHENNE MUSCULAR DYSTROPHY; GENETIC TRANSFECTION; GENETICS; HEART MUSCLE CELL; HUMAN; IMMUNOHISTOCHEMISTRY; INTRAMUSCULAR DRUG ADMINISTRATION; INTRAVENOUS DRUG ADMINISTRATION; METABOLISM; METHODOLOGY; MOUSE; WESTERN BLOTTING; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANIMALS; BLOTTING, WESTERN; CELL LINE; CELL-PENETRATING PEPTIDES; CELLS, CULTURED; DYSTROPHIN; HUMANS; IMMUNOHISTOCHEMISTRY; INJECTIONS, INTRAMUSCULAR; INJECTIONS, INTRAVENOUS; MICE; MICE, INBRED MDX; MUSCULAR DYSTROPHY, DUCHENNE; MYOCYTES, CARDIAC; OLIGONUCLEOTIDES, ANTISENSE; TRANSFECTION;

EID: 84859836034     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-61779-767-5_27     Document Type: Article

References (18)

1. Abes R, Arzumanov A, Moulton H, Abes S, Ivanova G, Gait MJ et al (2008) Arginine-rich cell penetrating peptides: design, structureactivity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides. J Pept Sci 14:455-460 (Pubitemid 351540906)
2. Wang Q, Yin H, Camelliti P, Betts C, Moulton H, Lee H et al (2010) In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy. J Gene Med 12:354-364
3. Ivanova GD, Arzumanov A, Abes R, Yin H, Wood MJ, Lebleu B et al (2008) Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 36:6418-6428
4. Yin H, Moulton HM, Betts C, Seow Y, Boutilier J 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
5. Wu B, Li Y, Morcos PA, Doran TJ, Lu P, Lu QL (2009) Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol Ther 17:864-871
6. 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)
7. Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S et al (2008) Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther 16:1624-1629
8. 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
9. Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ et al (2009) In vivo comparison of 2 ¢-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med 11:257-266
10. Moulton HM, Wu B, Jearawiriyapaisarn N, Sazani P, Lu QL, Kole R (2009) Peptide morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy. Ann N Y Acad Sci 1175:55-60
11. Yin H, Lu Q, Wood M (2008) Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 16:38-45
12. 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)
13. Lu QL, Rabinowitz A, Chen YC, Yokota T, Yin H, Alter J et al (2005) Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 102:198-203 (Pubitemid 40094452)
14. Malerba A, Thorogood FC, Dickson G, Graham IR (2009) Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther 20:955-965
15. Ivanova GD, Fabani MM, Arzumanov AA, Abes R, Yin H, Lebleu B et al (2008) PNApeptide conjugates as intracellular gene control agents. Nucleic Acids Symp Ser (Oxf) (52):31-32
16. Yin H, Saleh AF, Betts C, Camelliti P, Seow Y, Ashraf S et al (2011) Pip5 transductionpeptides direct high efficiency oligonucleotide-mediated dystrophin exonskipping in heart and phenotypic correction in mdxmice. Mol Ther 19(7):1295-1303
17. Seow Y, Yin H, Wood MJ (2010) Identification of a novel muscle targeting peptide in mdx mice. Peptides 31:1873-1877
18. Camelliti P, Gallagher JO, Kohl P, McCulloch AD (2006) Micropatterned cell cultures on elastic membranes as an in vitro model of myocardium. Nat Protoc 1:1379-1391