Pluronic-PEI copolymers enhance exon-skipping of 2′-O-methyl phosphorothioate oligonucleotide in cell culture and dystrophic mdx mice

Gene Therapy

Volumn 21, Issue 1, 2014, Pages 52-59

  Wang, M ^a   Wu, B ^a   Lu, P ^a   Tucker, J D ^a,b   Milazi, S ^a   Shah, S N ^a   Lu, Q L ^a  

^a Neuromuscular/ALS Center   (United States)

^b UNIVERSITY OF NORTH CAROLINA AT CHARLOTTE   (United States)

Author keywords

20 O methyl phosphorothioate RNA; amphiphilic polymers; antisense delivery; gene delivery; muscular dystrophy

Indexed keywords

2' O METHYL PHOSPHOROTHIOATE OLIGONUCLEOTIDE; AGAROSE; ANTISENSE OLIGONUCLEOTIDE; COPOLYMER; OLIGOMER; OLIGONUCLEOTIDE PHOSPHOROTHIOATE; PLASMID DNA; POLOXAMER; POLYETHYLENEIMINE DERIVATIVE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL CULTURE; CONTROLLED STUDY; DYSTROPHY; EXON SKIPPING; GENE TARGETING; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; IN VIVO GENE TRANSFER; IN VIVO STUDY; MOUSE; MUSCULAR DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; TIBIALIS ANTERIOR MUSCLE;

MUS;

ANIMALS; CELL LINE; DYSTROPHIN; EXONS; GENETIC THERAPY; INJECTIONS, INTRAMUSCULAR; LIPIDS; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; NANOPARTICLES; OLIGONUCLEOTIDES, ANTISENSE; PHOSPHOROTHIOATE OLIGONUCLEOTIDES; PLASMIDS; POLOXAMER; POLYETHYLENEIMINE;

EID: 84891739039     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2013.57     Document Type: Article

References (35)

1. Hoffman EP, Brown Jr RH, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919-928.
2. Koenig M, Beggs AH, Moyer M, Scherpf S, Heindrich K, Bettecken T et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet 1989; 45: 498-506. (Pubitemid 19261437)
3. Wu B, Moulton HM, Iversen PL, Jiang J, Li J, Spurney CF et al. Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci USA 2008; 105: 14814-14819.
4. Yin H, Moulton HM, Seow Y, Boyd C, Boutilier J, Iverson P et al. Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 2008; 17: 3909-3918. (Pubitemid 352762853)
5. Wu B, Lu P, Benrashid E, Malik S, Ashar J, Doran TJ et al. Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther 2009; 17: 132-140.
6. Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N et al. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 2011; 364: 1513-1522.
7. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 2011; 378: 595-605.
8. Sirsi SR, Schray RC, Guan X, Lykens NM, Williams JH, Erney ML et al. Functionalized PEG-PEI copolymers complexed to exon-skipping oligonucleotides improve dystrophin expression in mdx mice. Hum Gene Ther 2008; 19: 795-806.
9. Dubowitz V. A short history of the World Muscle Society. Neuromuscul Disord 2005; 15: 642-647. (Pubitemid 41139197)
10. Partridge TA. Stem cell route to neuromuscular therapies. Muscle Nerve 2003; 27: 133-141. (Pubitemid 36172466)
11. Wagner KR, Lechtzin N, Judge DP. Current treatment of adult Duchenne muscular dystrophy. Biochim Biophys Acta 2007; 1772: 229-237. (Pubitemid 46123913)
12. Townsend D, Yasuda S, Li S, Chamberlain JS, Metzger JM. Emergent dilated cardiomyopathy caused by targeted repair of dystrophic skeletal muscle. Mol Ther 2008; 16: 832-835. (Pubitemid 351587091)
13. Williams JH, Sirsi SR, Latta DR, Lutz GJ. Induction of dystrophin expression by exon-skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers. Mol Ther 2006; 14: 88-96. (Pubitemid 43922137)
14. Koo T, Wood. MJ. Clinical trials using antisense oligonucleotides in Duchenne muscular dystrophy. Hum Gene Ther 2013; 24: 479-488.
15. Eckstein F. Phosphorothioate oligodeoxynucleotides: what is their origin and what is unique about them Antisense Nucleic Acid Drug Dev 2000; 10: 117-121. (Pubitemid 30255596)
16. Kurreck J. Antisense technologies. Improvement through novel chemical modifications. Eur J Biochem 2003; 270: 1628-1644. (Pubitemid 36532522)
17. Chiappetta DA, Sosnik A. Poly(ethylene oxide)-poly(propylene oxide) block copolymer micelles as drug delivery agents: improved hydrosolubility, stability and bioavailability of drugs. Eur J Pharm Biopharm 2007; 66: 303-317. (Pubitemid 46756232)
18. Sriadibhatla S, Yang Z, Gebhart C, Alakhov VY, Kabanov A. Transcriptional activation of gene expression by pluronic block copolymers in stably and transiently transfected cells. Mol Ther 2006; 13: 804-813.
19. Pitard B, Pollard H, Agbulut O, Lambert O, Vilquin JT, Cherel Y et al. A nonionic amphiphile agent promotes gene delivery in vivo to skeletal and cardiac muscles. Hum Gene Ther 2002; 13: 1767-1775. (Pubitemid 35191750)
20. Roques C, Fromes Y, Fattal E. Hydrosoluble polymers for muscular gene delivery. Eur J Pharm Biopharm 2009; 72: 378-390.
21. Richard P, Bossard F, Desigaux L, Lanctin C, Bello-Roufai M, Pitard B. Amphiphilic block copolymers promote gene delivery in vivo to pathological skeletal muscles. Hum Gene Ther 2005; 16: 1318-1324. (Pubitemid 41611659)
22. Boussif O, Lezoualch F, Zanta MA, Mergny MD, Scherman D, Demeneix B et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad. Sci. USA 1995; 92: 7297-7301.
23. Lungwitz U, Breunig M, Blunk T, Gopferich A. Polyethylenimine-based non-viral gene delivery systems. Eur J Pharm Biopharm 2005; 60: 247-266. (Pubitemid 40804565)
24. Choosakoonkriang S, Lobo BA, Koe GS, Koe JG, Middaugh CR. Biophysical characterization of PEI/DNA complexes. J Pharm Sci 2003; 92: 1710-1722. (Pubitemid 36929690)
25. Garrett SW, Davies OR, Milroy DA, Wood PJ, Pouton CW, Threadgill MD. Synthesis and characterisation of polyamine-poly(ethylene glycol) constructs for DNA binding and gene delivery. Bioorg Med Chem 2000; 8: 1779-1797. (Pubitemid 30427245)
26. Gebhart CL, Sriadibhatla S, Vinogradov S, Lemieux P, Alakhov V, Kabanov AV. Design and formulation of polyplexes based on pluronic-polyethyleneimine conjugates for gene transfer. Bioconjug Chem 2002; 13: 937-944.
27. Wang MX, Lu P, Wu B, Tucker JD, Cloer C, Lu QL. High efficiency and low toxicity of polyethyleneimine modified pluronics (PEI-Pluronic) as gene delivery carriers in cell culture and dystrophic mdx mice. J Mater Chem 2012; 22: 6038-6046.
28. Wang MX, Wu B, Lu PJ, Cloer C, Tucker JD, Lu QL. Polyethylenimine modified pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx mice. Mol Ther 2013; 21: 210-216.
29. Grayson AC, Doody AM, Putnam D. Biophysical and structural characterization of polyethylenimine-mediated siRNA delivery in vitro. Pharm Res 2006; 23: 1868-1876. (Pubitemid 44200551)
30. Jääskeläinen I, Peltola S, Honkakoski P, Mönkkönen J, Urtti A. A lipid carrier with a membrane active component and a small complex size are required for efficient cellular delivery of anti-sense phosphorothioate oligonucleotides. Eur J Pharm Sci 2000; 10: 187-193. (Pubitemid 30190094)
31. Eliyahu H, Barenholz Y, Domb AJ. Polymers for DNA delivery. Molecules 2005; 10: 34-64. (Pubitemid 40404885)
32. Patrick M, Chantal P, Jean-Jacques Y, Paul-Alain J. Chemical vectors for gene delivery: a current review on polymers, peptides and lipids containing histidine or imidazole as nucleic acids carriers. Br J Pharmacol 2009; 157: 166-178.
33. Roques C, Bouchemal K, Ponchel G, Yves Fromes Y, Elias Fattal E. Parameters affecting organization and transfection efficiency of amphiphilic copolymers/DNA carriers. J Control Release 2009; 138: 71-77.
34. Sharma VK, Thomas M, Klibanov AM. Mechanistic studies on aggregation of polyethylenimine-DNA complexes and its prevention. Biotechnol Bioeng 2005; 90: 614-620. (Pubitemid 40733990)
35. Rimessi P, Sabatelli P, Fabris M, Braghetta P, Bassi E, Spitali P et al. Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse. Mol Ther 2009; 17: 820-827.