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.
Peter Sazani, Ryszard Kole
Title and authors | Publication | Year |
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Nuclear translocation of SIRT4 mediates deacetylation of U2AF2 to modulate renal fibrosis through alternative splicing-mediated upregulation of CCN2.
Yang G, Xiang J, Yang X, Liu X, Li Y, Li L, Kang L, Liang Z, Yang S |
eLife | 2024 |
Silencing of heat shock protein 90 (hsp90): Effect on development and infectivity of Ichthyophthirius multifiliis.
Saleh M, Abdel-Baki AS, Dkhil MA, El-Matbouli M, Al-Quraishy S |
BMC veterinary research | 2023 |
Pervasive occurrence of splice-site-creating mutations and their possible involvement in genetic disorders
Sakaguchi N, Suyama M |
npj Genomic Medicine | 2022 |
Fluorescence-Based Binding Characterization of Small Molecule Ligands Targeting CUG RNA Repeats.
Chang Z, Zheng YY, Mathivanan J, Valsangkar VA, Du J, Abou-Elkhair RAI, Hassan AEA, Sheng J |
International journal of molecular sciences | 2022 |
Targeting RNA structures with small molecules.
Childs-Disney JL, Yang X, Gibaut QMR, Tong Y, Batey RT, Disney MD |
Nature reviews. Drug discovery | 2022 |
Cell Penetrating Peptides: Methods and Protocols
Ü Langel |
2021 | |
Improving Strategies in the Development of Protein‐Downregulation‐Based Antiandrogens
R Zhang, C Huang, X Xiao, J Zhou |
ChemMedChem | 2021 |
Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias
R Afonso-Reis, IT Afonso, C Nóbrega |
International journal of molecular sciences | 2021 |
Clinical Reasoning: A 2-Day-Old Boy With Sudden Cardiac Arrest and Encephalopathy
BB Holmes, JB Russ, YW Wu, RC Gallagher, D Gano |
Neurology | 2021 |
Clinical Reasoning: A 2-Day-Old Boy With Sudden Cardiac Arrest and Encephalopathy.
Holmes BB, Russ JB, Wu YW, Gallagher RC, Gano D |
Neurology | 2021 |
Tumor suppressor genes and allele-specific expression: mechanisms and significance
EA Clayton, S Khalid, D Ban, L Wang, IK Jordan, JF McDonald |
Oncotarget | 2020 |
Gene Therapy for Hepatocellular Carcinoma: Highlighting the Journey from Theory to Clinical Applications
MA Younis, IA Khalil, H Harashima |
2020 | |
Cellular Targeting of Oligonucleotides by Conjugation with Small Molecules
M Hawner, C Ducho |
Molecules (Basel, Switzerland) | 2020 |
Nuclear and Cytoplasmatic Quantification of Unconjugated, Label-Free Locked Nucleic Acid Oligonucleotides
H Pendergraff, S Schmidt, J Vikeså, C Weile, C Øverup, MW Lindholm, T Koch |
Nucleic Acid Therapeutics (Formerly Oligonucleotides) | 2020 |
Exon skipping of TGFβRI affects signalling and ECM expression in hypertrophic scar-derived fibroblasts
RS Raktoe, MH Rietveld, JJ Out-Luiting, MK Julio, PP van Zuijlen, R van Doorn, AE Ghalbzouri |
2020 | |
Tumor suppressor genes and allele-specific expression: mechanisms and significance.
Clayton EA, Khalid S, Ban D, Wang L, Jordan IK, McDonald JF |
Oncotarget | 2020 |
Cellular Targeting of Oligonucleotides by Conjugation with Small Molecules.
Hawner M, Ducho C |
Molecules (Basel, Switzerland) | 2020 |
Suppression of prostate tumor cell survival by antisense oligonucleotide-mediated inhibition of AR-V7 mRNA synthesis
MV Velez, GW Verhaegh, F Smit, JP Sedelaar, JA Schalken |
Oncogene | 2019 |
RNA Secondary Structure-Based Design of Antisense Peptide Nucleic Acids for Modulating Disease-Associated Aberrant Tau Pre-mRNA Alternative Splicing
AA Ong, J Tan, M Bhadra, C Dezanet, KM Patil, MS Chong, R Kierzek, JL Decout, X Roca, G Chen |
Molecules (Basel, Switzerland) | 2019 |
Determination of the pathogenicity of known COL4A5 intronic variants by in vitro splicing assay
T Horinouchi, K Nozu, T Yamamura, S Minamikawa, C Nagano, N Sakakibara, K Nakanishi, Y Shima, N Morisada, S Ishiko, Y Aoto, H Nagase, H Takeda, R Rossanti, H Kaito, M Matsuo, K Iijima |
Scientific Reports | 2019 |
Downregulation of TdT Expression through Splicing Modulation by Antisense Peptide Nucleic Acid (PNA)
S Montazersaheb, M Kazemi, E Nabat, PE Nielsen, MS Hejazi |
Current pharmaceutical biotechnology | 2019 |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
Polyglutamine Disorders
C Nóbrega, LP de Almeida |
2018 | |
A cross-sectional study examining the expression of splice variants K-RAS4A and K-RAS4B in advanced non-small-cell lung cancer patients
V Aran, PM Domingues, FC de Macedo, CA de Sousa, TC Montella, MT de Accioly, CG Ferreira |
Lung Cancer | 2018 |
Alternative Splicing as a Target for Cancer Treatment
N Martinez-Montiel, N Rosas-Murrieta, MA Ruiz, E Monjaraz-Guzman, R Martinez-Contreras |
International journal of molecular sciences | 2018 |
In vitro inhibition of porcine reproductive and respiratory syndrome virus replication by short antisense oligonucleotides with locked nucleic acid modification
L Zhu, J Bi, L Zheng, Q Zhao, X Shu, G Guo, J Liu, G Yang, J Liu, G Yin |
BMC veterinary research | 2018 |
Modulation of MKNK2 alternative splicing by splice-switching oligonucleotides as a novel approach for glioblastoma treatment
M Mogilevsky, O Shimshon, S Kumar, A Mogilevsky, E Keshet, E Yavin, F Heyd, R Karni |
Nucleic Acids Research | 2018 |
Oligonucleotide-Based Drugs and Therapeutics: Preclinical and Clinical Considerations for Development
N Ferrari, R Seguin |
2018 | |
Potential of Peptide Nucleic Acids in Future Therapeutic Applications
S Montazersaheb, MS Hejazi, HN Charoudeh |
Advanced pharmaceutical bulletin | 2018 |
Recent progress on gene silencing/suppression by virus-derived small interfering RNAs in rice viruses especially Rice grassy stunt virus
M Arif, SU Islam, M Adnan, M Anwar, H Ali, Z Wu |
Microbial Pathogenesis | 2018 |
Alternative pre-mRNA splicing of Toll-like receptor signaling components in peripheral blood mononuclear cells from patients with ARDS
RZ Blumhagen, BR Hedin, KC Malcolm, EL Burnham, M Moss, E Abraham, TJ Huie, JA Nick, TE Fingerlin, S Alper |
American journal of physiology. Lung cellular and molecular physiology | 2017 |
Self-aggregating 1.8kDa polyethylenimines with dissolution switch at endosomal acidic pH are delivery carriers for plasmid DNA, mRNA, siRNA and exon-skipping oligonucleotides
M Chiper, N Tounsi, R Kole, A Kichler, G Zuber |
Journal of Controlled Release | 2017 |
Splicing regulatory factors, ageing and age-related disease
E Latorre, LW Harries |
Ageing Research Reviews | 2017 |
Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides
JK Bailey, W Shen, X Liang, ST Crooke |
Nucleic Acids Research | 2017 |
Alternative splicing in cancers: From aberrant regulation to new therapeutics
X Song, Z Zeng, H Wei, Z Wang |
Seminars in Cell & Developmental Biology | 2017 |
RNA Therapeutics in Oncology: Advances, Challenges, and Future Directions
AR MacLeod, ST Crooke |
The Journal of Clinical Pharmacology | 2017 |
The Role of Reactive Oxygen and Nitrogen Species in the Expression and Splicing of Nitric Oxide Receptor
IG Sharina, E Martin |
Antioxidants & Redox Signaling | 2016 |
Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity
CS Rocha, KE Lundin, MA Behlke, R Zain, SE Andaloussi, CI Smith |
Nucleic Acid Therapeutics (Formerly Oligonucleotides) | 2016 |
Correcting the NLRP3 inflammasome deficiency in macrophages from autoimmune NZB mice with exon skipping antisense oligonucleotides
SJ Thygesen, DP Sester, SO Cridland, SD Wilton, KJ Stacey |
Immunology and Cell Biology | 2016 |
Sodium Butyrate and Valproic Acid as Splicing Restoring Agents in Erythroid Cells of b-Thalassemic Patients
MS Khaniani, M Tagizadeh, AH Feizi, SM Derakhshan |
IRAN J BIOTECHNOL | 2016 |
Transposable element-driven transcript diversification and its relevance to genetic disorders
S Ayarpadikannan, HE Lee, K Han, HS Kim |
Gene | 2015 |
Conditional Control of Alternative Splicing through Light-Triggered Splice-Switching Oligonucleotides
J Hemphill, Q Liu, R Uprety, S Samanta, M Tsang, RL Juliano, A Deiters |
Journal of the American Chemical Society | 2015 |
Investigation of the effect of amino acid chirality in the internucleoside linker on DNA:DNA and DNA:RNA duplex stability
S Bagmare, AD Gunjal, VA Kumar |
Tetrahedron | 2015 |
Long noncoding RNAs and tumorigenesis: genetic associations, molecular mechanisms, and therapeutic strategies
F Zhang, L Zhang, C Zhang |
Tumor Biology | 2015 |
Antisense oligonucleotide induction of progerin in human myogenic cells
YB Luo, C Mitrpant, AM Adams, RD Johnsen, S Fletcher, FL Mastaglia, SD Wilton |
PloS one | 2014 |
Nanoparticle Delivery of Antisense Oligonucleotides and Their Application in the Exon Skipping Strategy for Duchenne Muscular Dystrophy
MS Falzarano, C Passarelli, A Ferlini |
Nucleic Acid Therapeutics (Formerly Oligonucleotides) | 2014 |
Porous Silicon–Cell Penetrating Peptide Hybrid Nanocarrier for Intracellular Delivery of Oligonucleotides
J Rytkönen, P Arukuusk, W Xu, K Kurrikoff, Ü Langel, VP Lehto, A Närvänen |
Molecular Pharmaceutics | 2014 |
Reference Module in Biomedical Sciences
DL Sackett, RB Haynes |
2014 | |
Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells
YB Luo, C Mitrpant, AM Adams, RD Johnsen, S Fletcher, FL Mastaglia, SD Wilton, T Preiss |
PloS one | 2014 |
Development of Artificial Nucleic Acids Functionalized for Damaged Gene Diagnosis, Gene Inhibition and Delivery System
O Nakagawa |
YAKUGAKU ZASSHI | 2014 |
Targeting the Splicing of mRNA in Autoimmune Diseases: BAFF Inhibition in Sjögren’s Syndrome as a Proof of Concept
N Roescher, JL Vosters, G Alsaleh, P Dreyfus, S Jacques, G Chiocchia, J Sibilia, PP Tak, JA Chiorini, X Mariette, JE Gottenberg |
Molecular Therapy | 2013 |
Direct intraventricular delivery of drugs to the rodent central nervous system
SL DeVos, TM Miller |
Journal of visualized experiments : JoVE | 2013 |
RNA and Cancer
JY Wu |
2013 | |
RNA and Cancer
JY Wu |
2013 | |
RNA and Cancer
JY Wu |
2013 | |
RNA and Cancer
JY Wu |
2013 | |
The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides
X Ming, K Carver, M Fisher, R Noel, JC Cintrat, D Gillet, J Barbier, C Cao, J Bauman, RL Juliano |
Nucleic Acids Research | 2013 |
Development of Novel Bioanalytical Methods to Determine the Effective Concentrations of Phosphorodiamidate Morpholino Oligomers in Tissues and Cells
FJ Schnell, SL Crumley, DV Mourich, PL Iversen |
BioResearch Open Access | 2013 |
Directing HER4 mRNA expression towards the CYT2 isoform by antisense oligonucleotide decreases growth of breast cancer cells in vitro and in vivo
TO Nielsen, S Sorensen, F Dagnæs-Hansen, J Kjems, BS Sorensen |
British Journal of Cancer | 2013 |
Antisense oligodeoxynucleotide inhibition as an alternative and convenient method for gene function analysis in pollen tubes
F Liao, L Wang, LB Yang, L Zhang, X Peng, MX Sun |
PloS one | 2013 |
Artificial Box C/D RNAs Affect Pre-mRNA Maturation in Human Cells
GA Stepanov, DV Semenov, AV Savelyeva, EV Kuligina, OA Koval, IV Rabinov, VA Richter |
BioMed Research International | 2013 |
HuR controls mitochondrial morphology through the regulation of Bcl xL translation
D Durie, M Hatzoglou, P Chakraborty, M Holcik |
Translation | 2013 |
Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
SL DeVos, TM Miller |
Journal of visualized experiments : JoVE | 2013 |
Synthesis of all four nucleoside-based β-amino acids as protected precursors for the synthesis of polyamide-DNA with alternating α-amino acid and nucleoside-β-amino acids
S Bagmare, M Varada, A Banerjee, VA Kumar |
Tetrahedron | 2013 |
Antisense Oligodeoxynucleotide Inhibition as an Alternative and Convenient Method for Gene Function Analysis in Pollen Tubes
F Liao, L Wang, LB Yang, L Zhang, X Peng, M Sun, GK Pandey |
PloS one | 2013 |
DNA and RNA Nanobiotechnologies in Medicine: Diagnosis and Treatment of Diseases
VA Erdmann, J Barciszewski |
2013 | |
Recent advances in gene therapy for thalassemia
JV Raja, MA Rachchh, RH Gokani |
Journal of Pharmacy & Bioallied Sciences | 2012 |
Alternative splicing: Functional diversity among voltage-gated calcium channels and behavioral consequences
D Lipscombe, A Andrade, SE Allen |
Biochimica et Biophysica Acta (BBA) - Biomembranes | 2012 |
The chemistry and biology of oligonucleotide conjugates
RL Juliano, X Ming, O Nakagawa |
Accounts of Chemical Research | 2012 |
Cell-penetrating Peptides as Versatile Vehicles for Oligonucleotide Delivery
H Margus, K Padari, M Pooga |
Molecular Therapy | 2012 |
Analogues of Artificial Human Box C/D Small Nucleolar RNA As Regulators of Alternative Splicing
of a pre-mRNA Target
GA Stepanov, DV Semenov, EV Kuligina, OA Koval, IV Rabinov, YY Kit, VA Richter |
Acta Naturae | 2012 |
Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy
GC Kendall, EI Mokhonova, M Moran, NE Sejbuk, DW Wang, O Silva, RT Wang, L Martinez, QL Lu, R Damoiseaux, MJ Spencer, SF Nelson, MC Miceli |
Science Translational Medicine | 2012 |
Tuning of alternative splicing--switch from proto-oncogene to tumor suppressor
A Shchelkunova, B Ermolinsky, M Boyle, I Mendez, M Lehker, KS Martirosyan, AV Kazansky |
International journal of biological sciences | 2012 |
Strategies toward well-defined polymer nanoparticles inspired by nature: Chemistry versus versatility
M Elsabahy, KL Wooley |
Journal of Polymer Science Part A Polymer Chemistry | 2012 |
Cell-penetrating peptides for the delivery of nucleic acids
T Lehto, K Kurrikoff, Ü Langel |
Expert Opinion on Drug Delivery | 2012 |
Cellular Uptake and Intracellular Trafficking of Antisense and siRNA Oligonucleotides
RL Juliano, X Ming, O Nakagawa |
Bioconjugate Chemistry | 2011 |
Antisense Oligonucleotides and RNA Interference
Kher G, Trehan S, Misra A |
Challenges in Delivery of Therapeutic Genomics and Proteomics | 2011 |
Encyclopedia of Life Sciences
B Dahlbäck |
Encyclopedia of Life Sciences | 2011 |
Progress in Molecular Biology and Translational Science
JF Wilkins, F Úbeda |
Modifications of Nuclear DNA and its Regulatory Proteins | 2011 |
Integrin targeted delivery of gene therapeutics
RL Juliano, X Ming, O Nakagawa, R Xu, H Yoo |
Theranostics | 2011 |
Effect of Steric Constraint at the γ-Backbone Position on the Conformations and Hybridization Properties of PNAs
MJ Crawford, S Rapireddy, R Bahal, I Sacui, DH Ly |
Journal of nucleic acids | 2011 |
PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation
K Ezzat, SE Andaloussi, EM Zaghloul, T Lehto, S Lindberg, PM Moreno, JR Viola, T Magdy, R Abdo, P Guterstam, R Sillard, SM Hammond, MJ Wood, AA Arzumanov, MJ Gait, CI Smith, M Hällbrink, Ü Langel |
Nucleic Acids Research | 2011 |
Modulation of biological processes in the nucleus by delivery of DNA oligonucleotides conjugated with gold nanoparticles
DW Kim, JH Kim, M Park, JH Yeom, H Go, S Kim, MS Han, K Lee, J Bae |
Biomaterials | 2011 |
Challenges in Delivery of Therapeutic Genomics and Proteomics
M Kumar, A Misra |
Challenges in Delivery of Therapeutic Genomics and Proteomics | 2011 |
Use of fully modified 2′-O-methyl antisense oligos for loss-of-function studies in vertebrate embryos
PN Schneider, JT Olthoff, AJ Matthews, DW Houston |
genesis | 2011 |
Oligonucleotide Analogs with Peptide Internucleotide Linkages
A Varizhuk, S Kochetkova, N Kolganova, E Timofeev, V Florentiev |
Nucleosides, Nucleotides and Nucleic Acids | 2011 |
Genetic therapies for RNA mis-splicing diseases
SM Hammond, MJ Wood |
Trends in Genetics | 2011 |
The role of carrier size in the pharmacodynamics of antisense and siRNA oligonucleotides
L Huang, B Sullenger, R Juliano |
Journal of Drug Targeting | 2010 |
Cell-Penetrating Peptides
Ü Langel |
Cell-Penetrating Peptides | 2010 |
Cell-Penetrating Peptides
Ü Langel |
Cell-Penetrating Peptides | 2010 |
Cell-Penetrating Peptides
Ü Langel |
Cell-Penetrating Peptides | 2010 |
Cell-Penetrating Peptides
Ü Langel |
Cell-Penetrating Peptides | 2010 |
Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases
L Du, RA Gatti |
Journal of Immunological Methods | 2010 |
Targeted Intracellular Delivery of Antisense Oligonucleotides via Conjugation with Small-Molecule Ligands
O Nakagawa, X Ming, L Huang, RL Juliano |
Journal of the American Chemical Society | 2010 |
The Biological Effect of an Antisense Oligonucleotide Depends on Its Route of Endocytosis and Trafficking
MR Alam, X Ming, V Dixit, M Fisher, X Chen, RL Juliano |
Oligonucleotides | 2010 |
K-rasexon 4A has a tumour suppressor effect on carcinogen-induced murine colonic adenoma formation
F Luo, H Ye, R Hamoudi, G Dong, W Zhang, CE Patek, G Poulogiannis, MJ Arends |
The Journal of Pathology | 2010 |
Prognostic significance of TRAIL death receptors in Middle Eastern colorectal carcinomas and their correlation to oncogenic KRAS alterations
P Bavi, SE Prabhakaran, J Abubaker, Z Qadri, T George, N Al-Sanea, A Abduljabbar, LH Ashari, S Alhomoud, F Al-Dayel, AR Hussain, S Uddin, KS Al-Kuraya |
Molecular Cancer | 2010 |
Pharmaceutical Sciences Encyclopedia: Drug Discovery, Development, and Manufacturing
CD Davis, UM Hanumegowda |
Pharmaceutical Sciences Encyclopedia: Drug Discovery, Development, and Manufacturing | 2010 |
Antisense Modulation of Both Exonic and Intronic Splicing Motifs Induces Skipping of a DMD Pseudo-Exon Responsible for X-Linked Dilated Cardiomyopathy
P Rimessi, M Fabris, M Bovolenta, E Bassi, S Falzarano, F Gualandi, C Rapezzi, F Coccolo, D Perrone, A Medici, A Ferlini |
Human Gene Therapy | 2010 |
Future alternative therapies for β-thalassemia
S Rivella, E Rachmilewitz |
Expert Review of Hematology | 2009 |
Gene therapy in thalassemia and hemoglobinopathies
L Breda, R Gambari, S Rivella |
Mediterranean journal of hematology and infectious diseases | 2009 |
RNA and Disease
TA Cooper, L Wan, G Dreyfuss |
Cell | 2009 |
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
S Svasti, T Suwanmanee, S Fucharoen, HM Moulton, MH Nelson, N Maeda, O Smithies, R Kole |
Proceedings of the National Academy of Sciences | 2009 |
A deep intronic mutation inFGBcreates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment
RL Davis, VM Homer, PM George, SO Brennan |
Human Mutation | 2009 |
Progress toward therapy with antisense-mediated splicing modulation
L Du, RA Gatti |
Current opinion in molecular therapeutics | 2009 |
Polypyrimidine tract binding protein prevents activity of an intronic regulatory element that promotes usage of a composite 3'-terminal exon
V Anquetil, CL Sommer, A Méreau, S Hamon, H Lerivray, S Hardy |
The Journal of biological chemistry | 2009 |
Modification of Alternative Splicing of Mcl-1 Pre-mRNA Using Antisense Morpholino Oligonucleotides Induces Apoptosis in Basal Cell Carcinoma Cells
JJ Shieh, KT Liu, SW Huang, YJ Chen, TY Hsieh |
Journal of Investigative Dermatology | 2009 |
Prognostic significance of alterations in KRAS isoforms KRAS-4A/4B and KRAS mutations in colorectal carcinoma
J Abubaker, P Bavi, W Al-Haqawi, M Sultana, S Al-Harbi, N Al-Sanea, A Abduljabbar, LH Ashari, S Alhomoud, F Al-Dayel, S Uddin, KS Al-Kuraya |
The Journal of Pathology | 2009 |
Effect of chirality of l/d-proline and prochiral glycine as the linker amino acid in five-atom linked thymidinyl-(α-amino acid)-thymidine dimers
S Bagmare, M D’Costa, VA Kumar |
Chemical Communications | 2009 |
Alternative splicing and disease
J Tazi, N Bakkour, S Stamm |
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease | 2009 |
Polypyrimidine Tract Binding Protein Prevents Activity of an Intronic Regulatory Element That Promotes Usage of a Composite 3′-Terminal Exon
V Anquetil, CL Sommer, A Méreau, S Hamon, H Lerivray, S Hardy |
The Journal of biological chemistry | 2009 |
Handbook of Research on Systems Biology Applications in Medicine:
A Daskalaki |
2009 | |
Recognition Receptors in Biosensors
M Zourob |
2009 | |
Formulation of polylactide-co-glycolic acid nanospheres for encapsulation and sustained release of poly(ethylene imine)-poly(ethylene glycol) copolymers complexed to oligonucleotides
SR Sirsi, RC Schray, MA Wheatley, GJ Lutz |
Journal of nanobiotechnology | 2009 |
Antisense Makes Sense in Engineered Regenerative Medicine
Y Yao, C Wang, RR Varshney, DA Wang |
Pharmaceutical Research | 2008 |
Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides
R Juliano, MR Alam, V Dixit, H Kang |
Nucleic Acids Research | 2008 |
Alternative splicing and tumor progression
C Ghigna, C Valacca, G Biamonti |
Current genomics | 2008 |
Peptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: quantitative evaluation of overall efficacy employing easy to handle reporter systems
SD Laufer, T Restle |
Current pharmaceutical design | 2008 |
Functional characterization of alternatively spliced human SECISBP2 transcript variants
LV Papp, J Wang, D Kennedy, D Boucher, Y Zhang, VN Gladyshev, RN Singh, KK Khanna |
Nucleic Acids Research | 2008 |
Dynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides
KB Wee, ZA Pramono, JL Wang, KF MacDorman, PS Lai, WC Yee |
PloS one | 2008 |
Chimeric (α-amino acid + nucleoside-β-amino acid)n peptide oligomers show sequence specific DNA/RNA recognition
K Gogoi, VA Kumar |
Chemical Communications | 2008 |
Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes
J Marquis, SS Kämpfer, L Angehrn, D Schümperli |
Gene Therapy | 2008 |
Effective delivery of antisense peptide nucleic acid oligomers into cells by anthrax protective antigen
DG Wright, Y Zhang, JR Murphy |
Biochemical and Biophysical Research Communications | 2008 |
Controlled delivery of anti-sense oligodeoxynucleotide from multilayered biocompatible phosphorylcholine polymer films
Z Zhang, X Cao, X Zhao, CM Holt, AL Lewis, JR Lu |
Journal of Controlled Release | 2008 |
Design and development of antisense drugs
R Malik, I Roy |
Expert Opinion on Drug Discovery | 2008 |
Integrated DNA, cDNA, and protein studies in Becker muscular dystrophy show high exception to the reading frame rule
A Kesari, LN Pirra, L Bremadesam, O McIntyre, E Gordon, AL Dubrovsky, V Viswanathan, EP Hoffman |
Human Mutation | 2008 |
COMPARATIVE ANALYSIS OF DISEASE-ASSOCIATED MUTATIONS IN THE CODING REGIONS OF ALTERNATIVELY AND CONSTITUTIVELY SPLICED HUMAN GENES
Q Huang, Y Li, J Li-Ling, H Huang, X Chen, F Ma |
Journal of Biological Systems | 2008 |
Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides
KB Wee, ZA Pramono, JL Wang, KF MacDorman, PS Lai, WC Yee, JN Volff |
PloS one | 2008 |
Splice-switching efficiency and specificity for oligonucleotides with locked nucleic acid monomers
P Guterstam, M Lindgren, H Johansson, U Tedebark, J Wengel, SE Andaloussi, Ü Langel |
Biochemical Journal | 2008 |
Distinct Uptake Routes of Cell-Penetrating Peptide Conjugates
P Lundin, H Johansson, P Guterstam, T Holm, M Hansen, Ü Langel, SE Andaloussi |
Bioconjugate Chemistry | 2008 |
Factors Determining the Efficacy of Nuclear Delivery of Antisense Oligonucleotides by Gold Nanoparticles
Y Liu, S Franzen |
Bioconjugate Chemistry | 2008 |
Cellular Delivery and Biological Activity of Antisense Oligonucleotides Conjugated to a Targeted Protein Carrier
H Kang, R Alam, V Dixit, M Fisher, RL Juliano |
Bioconjugate Chemistry | 2008 |
Antisense oligonucleotide-induced alternative splicing of the APOB mRNA generates a novel isoform of APOB
B Khoo, X Roca, SL Chew, AR Krainer |
BMC Molecular Biology | 2007 |
New biological therapies from the human genome
S Schnieper-Samec, G Feger, TN Wells |
Expert Opinion on Drug Discovery | 2007 |
Alternative splicing: an important mechanism for myometrial gene regulation that can be manipulated to target specific genes associated with preterm labour
AJ Tyson-Capper |
BMC pregnancy and childbirth | 2007 |
Switching on transgene expression by correcting aberrant splicing using multi-targeting steric-blocking oligonucleotides
S Resina, R Kole, A Travo, B Lebleu, AR Thierry |
The Journal of Gene Medicine | 2007 |
A Novel Cell-penetrating Peptide, M918, for Efficient Delivery of Proteins and Peptide Nucleic Acids
S El-Andaloussi, HJ Johansson, T Holm, Ü Langel |
Molecular Therapy | 2007 |
Assessing the delivery efficacy and internalization route of cell-penetrating peptides
SE Andaloussi, P Guterstam, Ü Langel |
Nature Protocols | 2007 |
Alternative splicing: an emerging topic in molecular and clinical oncology
MJ Pajares, T Ezponda, R Catena, A Calvo, R Pio, LM Montuenga |
The Lancet Oncology | 2007 |
Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos
PA Morcos |
Biochemical and Biophysical Research Communications | 2007 |
RNA regulation and cancer development
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 |
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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
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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 |
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Peptide functionalized nanoparticles for nonviral gene delivery
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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
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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 |