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Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing

Mariano A. Garcia-Blanco

Mariano A. Garcia-Blanco

Published August 15, 2003
Citation Information: J Clin Invest. 2003;112(4):474-480. https://doi.org/10.1172/JCI19462.

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Citations to this article (61)

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Mutation-Directed Therapeutics for Neurofibromatosis Type I A Leier, DM Bedwell, AT Chen, G Dickson, KM Keeling, RA Kesterson, BR Korf, TT Lago, UF Müller, L Popplewell, J Zhou, D Wallis	Molecular Therapy — Nucleic Acids	2020
The Role of Number of Copies, Structure, Behavior and Copy Number Variations (CNV) of the Y Chromosome in Male Infertility F Signore, C Gulìa, R Votino, VD Leo, S Zaami, L Putignani, S Gigli, E Santini, L Bertacca, A Porrello, R Piergentili	Genes & development	2019
SHIP1 Deficiency in Inflammatory Bowel Disease Is Associated With Severe Crohn’s Disease and Peripheral T Cell Reduction S Fernandes, N Srivastava, R Sudan, FA Middleton, AK Shergill, JC Ryan, WG Kerr	Frontiers in immunology	2018
Molecular barcoding of viral vectors enables mapping and optimization of mRNA trans -splicing M Davidsson, P Díaz-Fernández, M Torroba, OD Schwich, P Aldrin-Kirk, L Quintino, A Heuer, G Wang, C Lundberg, T Björklund	RNA (New York, N.Y.)	2018
RNA Structure Design Improves Activity and Specificity of trans -Splicing-Triggered Cell Death in a Suicide Gene Therapy Approach S Poddar, PS Loh, ZH Ooi, F Osman, J Eul, V Patzel	Molecular Therapy — Nucleic Acids	2018
Optimization of trans-Splicing for Huntington's Disease RNA Therapy H Rindt, CM Tom, CL Lorson, VB Mattis	Frontiers in neuroscience	2017
De novo assembly and characterization of breast cancer transcriptomes identifies large numbers of novel fusion-gene transcripts of potential functional significance VK Mittal, JF McDonald	BMC Medical Genomics	2017
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IL-33 isoforms: their future as vaccine adjuvants? DO Villarreal, DB Weiner	Expert Review of Vaccines	2015
Design and Characterization of a Twin Ribozyme for Potential Repair of a Deletion Mutation within the Oncogenic CTNNB1 -ΔS45 mRNA D Balke, I Zieten, A Strahl, O Müller, S Müller	ChemMedChem	2014
α1-Antitrypsin Deficiency D Perlmutter	Zakim and Boyer s Hepatology	2012
IL-7 splicing variant IL-7δ5 induces human breast cancer cell proliferation via activation of PI3K/Akt pathway D Pan, B Liu, X Jin, J Zhu	Biochemical and Biophysical Research Communications	2012
Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns M Shababi, CL Lorson	Journal of Molecular Neuroscience	2011
RNA reprogramming and repair based on trans-splicing group I ribozymes T Fiskaa, ÅB Birgisdottir	New Biotechnology	2010
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Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy TH Coady, CL Lorson	The Journal of neuroscience : the official journal of the Society for Neuroscience	2010
Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts S Lorain, C Peccate, ML Hir, L Garcia, A Lewin	PloS one	2010
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Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing T Rodriguez-Martin, K Anthony, MA Garcia-Blanco, SG Mansfield, BH Anderton, JM Gallo	Human Molecular Genetics	2009
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Expression analysis and functional activity of interleukin-7 splice variants NK Vudattu, I Magalhaes, H Hoehn, D Pan, MJ Maeurer	Genes and Immunity	2008
Sequestering of the 3′ Splice Site in a Theophylline-Responsive Riboswitch Allows Ligand-Dependent Control of Alternative Splicing V Gusti, DS Kim, RK Gaur	Oligonucleotides	2008
Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo J Wang, SG Mansfield, CA Cote, PD Jiang, K Weng, MJ Amar, BH Brewer, AT Remaley, GJ McGarrity, MA Garcia-Blanco, M Puttaraju	Molecular Therapy	2008
Development of a single vector system that enhances trans-splicing of SMN2 transcripts TH Coady, TD Baughan, M Shababi, MA Passini, CL Lorson	PloS one	2008
Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts TH Coady, TD Baughan, M Shababi, MA Passini, CL Lorson, J Valcarcel	PloS one	2008
Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA Splicing TH Coady, M Shababi, GE Tullis, CL Lorson	Molecular Therapy	2007
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Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector T Baughan, M Shababi, TH Coady, AM Dickson, GE Tullis, CL Lorson	Molecular Therapy	2006
RNA interference: a potential therapeutic tool for silencing splice isoforms linked to human diseases RK Gaur	BioTechniques	2006
Alternative Splicing and Disease P Jeanteur		2006
Splicing regulators: targets and drugs GW Yeo	Genome biology	2005
Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies T Rodriguez-Martin, MA Garcia-Blanco, SG Mansfield, AC Grover, M Hutton, Q Yu, J Zhou, BH Anderton, JM Gallo	Proceedings of the National Academy of Sciences	2005
Selective modification of alternative splicing by indole derivatives that target serine-arginine-rich protein splicing factors J Soret, N Bakkour, S Maire, S Durand, L Zekri, M Gabut, W Fic, G Divita, C Rivalle, D Dauzonne, CH Nguyen, P Jeanteur, J Tazi	Proceedings of the National Academy of Sciences	2005
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Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing S Bhaumik, Z Walls, M Puttaraju, LG Mitchell, SS Gambhir	Proceedings of the National Academy of Sciences	2004
When proteome meets genome: the alpha helix and the beta strand of proteins are eschewed by mRNA splice junctions and may define the minimal indivisible modules of protein architecture S Barik	Journal of Biosciences	2004
Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency M Tahara, RG Pergolizzi, H Kobayashi, A Krause, K Luettich, ML Lesser, RG Crystal	Nature Medicine	2004
RNA repair using spliceosome-mediated RNA trans-splicing SG Mansfield, H Chao, CE Walsh	Trends in Molecular Medicine	2004
New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies Christopher E Walsh	Seminars in Hematology	2004
Molecular Cloning and Characterization of Two Mouse Peroxisome Proliferator-activated Receptor α (PPARα)-regulated Peroxisomal Acyl-CoA Thioesterases MA Westin, SE Alexson, MC Hunt	The Journal of biological chemistry	2004
Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements IG Bruno, W Jin, GJ Cote	Human Molecular Genetics	2004
Alternative splicing in disease and therapy MA Garcia-Blanco, AP Baraniak, EL Lasda	Nature Biotechnology	2004
Ribozyme-mediated revision of RNA and DNA MB Long, JP Jones, BA Sullenger, J Byun	Journal of Clinical Investigation	2003
Targeted genetic repair: an emerging approach to genetic therapy BA Sullenger	Journal of Clinical Investigation	2003
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