Splice Redirection as a Convenient Assay to Monitor CPP–ON Efficiency and Mechanism

Methods in Molecular Biology

Volumn 683, Issue , 2011, Pages 307-320

  Abes, Rachida ^a   Arzumanov, Andrey A ^b   Saleh, Amer F ^b   Hassane, Fatouma Said ^a   Gait, Michael J ^b   Lebleu, Bernard ^a  

^a UNIV MONTPELLIER   (France)

^b MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

Author keywords

Cell penetrating peptides; Delivery; Liposome leakage; Oligonucleotides; Splicing regulation

Indexed keywords

CELL PENETRATING PEPTIDE; DISULFIDE; DRUG CARRIER; LIPOSOME; LUCIFERASE; MALEIMIDE; MALEIMIDE DERIVATIVE; OLIGODEOXYRIBONUCLEOTIDE; OLIGONUCLEOTIDE; PEPTIDE NUCLEIC ACID; SAPONIN;

ARTICLE; CELL MEMBRANE PERMEABILITY; CHEMISTRY; ENDOCYTOSIS; FLOW CYTOMETRY; GENETICS; HELA CELL; HUMAN; METABOLISM; RNA SPLICING;

CELL MEMBRANE PERMEABILITY; CELL-PENETRATING PEPTIDES; DISULFIDES; DRUG CARRIERS; ENDOCYTOSIS; FLOW CYTOMETRY; HELA CELLS; HUMANS; LIPOSOMES; LUCIFERASES; MALEIMIDES; OLIGODEOXYRIBONUCLEOTIDES; OLIGONUCLEOTIDES; PEPTIDE NUCLEIC ACIDS; RNA SPLICING; SAPONINS;

MLCS; MLOWN;

EID: 79952198482     PISSN: 10643745     EISSN: 19406029     Source Type: Book Series    
DOI: 10.1007/978-1-60761-919-2_22     Document Type: Chapter

References (9)

1. Bauman, J., Jearawiriyapaisam N. and Kole, R. (2009) Therapeutic potential of splice-switching oligonucleotides. Oligonucleotides 19, 1–13.
2. Wilton, S.D. and Fletcher, S. (2008) Exon skipping and Duchenne muscular dystrophy: Hope, hype and how feasible. Neurol. India 56, 254–262.
3. Kang, S.H., Cho, M.J. and Kole, R. (1998) Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry 37, 6235–6239.
4. Abes, S., Turner, J.J., Abes, R., Arzumanov, A.A., Williams, D, Owen, D, Lebleu, B. and Gait, M.J. (2009) Peptide-based delivery of steric-block PNA oligonucleotides. Methods Mol. Biol. 480, 85–99.
5. Richard, J.P., Melikov, K., Vives, E., Ramos, C., Verbeure, B., Gait, M.J., Chernomordik, L.V. and Lebleu, B. (2003) Cell-penetrating peptides. A re-evaluation of the mechanism of cellular uptake. J. Biol. Chem. 278, 585–590.
6. Turner, J.J., Ivanova, G.D., Verbeure, B, Williams, D., Arzumanov, A.A., Abes, S., Lebleu, B. and Gait, M.J. (2005) Cell-penetrating peptide conjugates of peptide nucleic acids (PNA) as inhibitors of HIV-1 Tat-dependent trans-activation in cells. Nucleic Acids Res. 33, 6837–6849.
7. Abes, S., Turner, J.J., Owen, D., Ivanova, G.D., Verbeure, B., Williams, D., Arzumanov, A.A., Clair, P., Gait, M.J. and Lebleu, B. (2007) Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide. Nucleic Acids Res. 35, 4495–4502.
8. Ivanova, G.D., 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.
9. Abes, S., 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.