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Citations to this article

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
Peter Sazani, Ryszard Kole
Peter Sazani, Ryszard Kole
Published August 15, 2003
Citation Information: J Clin Invest. 2003;112(4):481-486. https://doi.org/10.1172/JCI19547.
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Perspective Series Article has an altmetric score of 15

Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

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Abstract

An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.

Authors

Peter Sazani, Ryszard Kole

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Year: 2024 2023 2022 2021 2020 2019 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 2008 2007 2006 2005 2004 2003 Total
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E Scholzová, R Malík, J Ševčík, Z Kleibl
Cancer Letters 2007
Pegylation of liposomes favours the endosomal degradation of the delivered phosphodiester oligonucleotides
K Remaut, B Lucas, K Braeckmans, J Demeester, SC Smedt
Journal of Controlled Release 2007
Lipoplex and peptide-based strategies for the delivery of steric-block oligonucleotides
S Resina, S Abes, JJ Turner, P Prevot, A Travo, P Clair, MJ Gait, AR Thierry, B Lebleu
International Journal of Pharmaceutics 2007
Alternative Splicing in the Postgenomic Era
BJ Blencowe, BR Graveley
2007
Synthesis and RNA Binding Selectivity of Oligonucleotides Modified with Five-Atom Thioacetamido Nucleic Acid Backbone Structures
K Gogoi, AD Gunjal, UD Phalgune, VA Kumar
Organic letters 2007
Applications of cell-penetrating peptides in regulation of gene expression
P Järver, K Langel, S El-Andaloussi, Ü Langel
Biochemical Society Transactions 2007
Pharmacokinetics, Biodistribution, Stability and Toxicity of a Cell-Penetrating Peptide−Morpholino Oligomer Conjugate
A Amantana, HM Moulton, ML Cate, MT Reddy, T Whitehead, JN Hassinger, DS Youngblood, PL Iversen
Bioconjugate Chemistry 2007
Defective splicing, disease and therapy: searching for master checkpoints in exon definition
E Buratti, M Baralle, FE Baralle
Nucleic Acids Research 2006
TassDB: a database of alternative tandem splice sites
M Hiller, S Nikolajewa, K Huse, K Szafranski, P Rosenstiel, S Schuster, R Backofen, M Platzer
Nucleic Acids Research 2006
Human laminopathies: nuclei gone genetically awry
BC Capell, FS Collins
Nature Reviews Genetics 2006
Comparison of basic peptides- and lipid-based strategies for the delivery of splice correcting oligonucleotides
AR Thierry, S Abes, S Resina, A Travo, JP Richard, P Prevot, B Lebleu
Biochimica et Biophysica Acta (BBA) - Biomembranes 2006
Analysis of Prostate-Specific Membrane Antigen Splice Variants in LNCap Cells
T Williams, R Kole
Oligonucleotides 2006
Induction of splice correction by cell-penetrating peptide nucleic acids
S El-Andaloussi, HJ Johansson, P Lundberg, Ü Langel
The Journal of Gene Medicine 2006
Efficient and Persistent Splice Switching by Systemically Delivered LNA Oligonucleotides in Mice
J ROBERTS, E PALMA, P SAZANI, H ORUM, M CHO, R KOLE
Molecular Therapy 2006
Modulation of calcium signalling by dominant negative splice variant of ryanodine receptor subtype 3 in native smooth muscle cells
F Dabertrand, JL Morel, V Sorrentino, J Mironneau, C Mironneau, N Macrez
Cell Calcium 2006
Alternative Splicing and Disease
P Jeanteur
2006
Modification of MyD88 mRNA Splicing and Inhibition of IL-1β Signaling in Cell Culture and in Mice with a 2′- O -Methoxyethyl-Modified Oligonucleotide
TA Vickers, H Zhang, MJ Graham, KM Lemonidis, C Zhao, NM Dean
Journal of immunology (Baltimore, Md. : 1950) 2006
Overexpression of Caspase-3s Splice Variant in Locally Advanced Breast Carcinoma Is Associated with Poor Response to Neoadjuvant Chemotherapy
F Végran, R Boidot, C Oudin, JM Riedinger, F Bonnetain, S Lizard-Nacol
Clinical cancer research 2006
Splicing of a Critical Exon of Human Survival Motor Neuron Is Regulated by a Unique Silencer Element Located in the Last Intron
NK Singh, NN Singh, EJ Androphy, RN Singh
Molecular and cellular biology 2006
Delivery of Phosphodiester Oligonucleotides: Can DOTAP/DOPE Liposomes Do the Trick?
K Remaut, B Lucas, K Braeckmans, NN Sanders, J Demeester, SC Smedt
Biochemistry 2006
Epigenetic manipulation of gene expression: a toolkit for cell biologists
RL Juliano, VR Dixit, H Kang, TY Kim, Y Miyamoto, D Xu
The Journal of Cell Biology 2005
Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics
D Gauguier
Encyclopedia of Genetics, Genomics, Proteomics and Bioinformatics 2005
Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome
P Scaffidi, T Misteli
Nature Medicine 2005
Predicting RNA secondary structure by free energy minimization
DH Mathews
Theoretical Chemistry Accounts 2005
Antibodies as Drug Carriers III: Design of Oligonucleotides with Enhanced Binding Affinity for Immunoglobulin G
E Palma, DG Klapper, MJ Cho
Pharmaceutical Research 2005
Avery's Diseases of the Newborn
R Chmait, TR Moore
Avery's Diseases of the Newborn 2005
Protection of oligonucleotides against nucleases by pegylated and non-pegylated liposomes as studied by fluorescence correlation spectroscopy
K Remaut, B Lucas, K Braeckmans, NN Sanders, J Demeester, SC Smedt
Journal of Controlled Release 2005
Alternative splicing: A new drug target of the post-genome era
M Hagiwara
Biochimica et Biophysica Acta (BBA) - Proteins & Proteomics 2005
Regulation of Apoptosis by Alternative Pre-mRNA Splicing
C Schwerk, K Schulze-Osthoff
Molecular Cell 2005
Use of Antisense Oligonucleotides in Functional Genomics and Target Validation
LV Ravichandran, NM Dean, EG Marcusson
Oligonucleotides 2004
Modification of Alternative Splicing by Antisense Therapeutics
R Kole, M Vacek, T Williams
Oligonucleotides 2004
Libraries enriched for alternatively spliced exons reveal splicing patterns in melanocytes and melanomas
A Watahiki, K Waki, N Hayatsu, T Shiraki, S Kondo, M Nakamura, D Sasaki, T Arakawa, J Kawai, M Harbers, Y Hayashizaki, P Carninci
Nature Methods 2004
Targeted genetic repair: an emerging approach to genetic therapy
BA Sullenger
Journal of Clinical Investigation 2003
Mending the message
MA Garcia-Blanco
Nature Biotechnology 2003
RNA directed therapeutics: mechanisms and status
ST Crooke
Drug Discovery Today: Therapeutic Strategies
Peptide functionalized nanoparticles for nonviral gene delivery
RM Levine, CM Scott, E Kokkoli
Soft Matter 2013
Oligonucleotides containing a peptide internucleotide linkage. A novel class of modified oligonucleotides
AM Varizhuk, SV Kochetkova, NA Kolganova, EN Timofeev, VL Florentiev
Russian Journal of Bioorganic Chemistry 2010
Spliceosome-Mediated RNA Trans-splicing
Y Yang, CE Walsh
Molecular Therapy 2005
Alternatively and Constitutively Spliced Exons Are Subject to Different Evolutionary Forces
FC Chen, SS Wang, CJ Chen, WH Li, TJ Chuang
Molecular Biology and Evolution 2005
Identification and characterization of a novel gene Saf transcribed from the opposite strand of Fas
MD Yan, CC Hong, GM Lai, AL Cheng, YW Lin, SE Chuang
Human Molecular Genetics 2005
Novel Targeting of Cyclooxygenase-2 (COX-2) Pre-mRNA Using Antisense Morpholino Oligonucleotides Directed to the 3′ Acceptor and 5′ Donor Splice Sites of Exon 4: Suppression of COX-2 Activity in Human Amnion-Derived WISH and Myometrial Cells
AJ Tyson-Capper, GN Europe-Finner
Molecular pharmacology 2005
Physiochemical Properties of Low and High Molecular Weight Poly(ethylene glycol)-Grafted Poly(ethylene imine) Copolymers and Their Complexes with Oligonucleotides
M Glodde, SR Sirsi, GJ Lutz
Biomacromolecules 2005
Épissage alternatif, pathologie et thérapeutique moléculaire
L Corcos, S Solier
médecine/sciences 2005
Antibodies as drug carriers III: design of oligonucleotides with enhanced binding affinity for immunoglobulin G.
Palma E, Klapper DG, Cho MJ
Pharmaceutical Research 2005
Androgen receptor function is modulated by the tissue-specific AR45 variant: Androgen receptor variant form
I Ahrens-Fath, O Politz, C Geserick, B Haendler
FEBS Journal 2004
New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies
Christopher E Walsh
Seminars in Hematology 2004
Manipulation of Alternative Splicing by a Newly Developed Inhibitor of Clks
M Muraki, B Ohkawara, T Hosoya, H Onogi, J Koizumi, T Koizumi, K Sumi, J Yomoda, MV Murray, H Kimura, K Furuichi, H Shibuya, AR Krainer, M Suzuki, M Hagiwara
The Journal of biological chemistry 2004
Alternative splicing in disease and therapy
MA Garcia-Blanco, AP Baraniak, EL Lasda
Nature Biotechnology 2004
Structural Determinants for High-Affinity Binding of Insulin-Like Growth Factor II to Insulin Receptor (IR)-A, the Exon 11 Minus Isoform of the IR
A Denley, ER Bonython, GW Booker, LJ Cosgrove, BE Forbes, CW Ward, JC Wallace
Molecular Endocrinology 2004
Selective Inhibition of Topoisomerase I and Various Steps of Spliceosome Assembly by Diospyrin Derivatives
J Tazi, N Bakkour, J Soret, L Zekri, B Hazra, W Laine, B Baldeyrou, A Lansiaux, C Bailly
Molecular pharmacology 2004
Grundlagen der Molekularen Medizin
D Ganten, K Ruckpaul
2003

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