Overview on applications of antisense-mediated exon skipping.

No abstract is available for this paper.

• Publication year

  2012

• Venue

  Methods in molecular biology

• Publication date

  Unknown publication date

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1007/978-1-61779-767-5_6PMID 22454056
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Modulation of biological processes in the nucleus by delivery of DNA oligonucleotides conjugated with gold nanoparticles.

  2011cited by this paper
• Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

  2011cited by this paper
• Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

  2011cited by this paper
• Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.

  2011cited by this paper
• Therapeutic exon ‘switching’ for dysferlinopathies?

  2010cited by this paper
• Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy

  2010cited by this paper
• Therapeutic exon skipping for dysferlinopathies?

  2010cited by this paper
• Efficient bypass of mutations in dysferlin deficient patient cells by antisense‐induced exon skipping

  2010cited by this paper
• Repair of aberrant splicing in growth hormone receptor by antisense oligonucleotides targeting the splice sites of a pseudoexon.

  2010cited by this paper
• Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

  2010cited by this paper
• PNA-mediated modulation and redirection of Her-2 pre-mRNA splicing: specific skipping of erbB-2 exon 19 coding for the ATP catalytic domain.

  2010cited by this paper
• Activating the synthesis of progerin, the mutant prelamin A in Hutchinson–Gilford progeria syndrome, with antisense oligonucleotides

  2009cited by this paper
• Functional analysis of three splicing mutations identified in the PMM2 gene: Toward a new therapy for congenital disorder of glycosylation type Ia

  2009cited by this paper
• Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation.

  2009cited by this paper
• Modification of alternative splicing of Mcl-1 pre-mRNA using antisense morpholino oligonucleotides induces apoptosis in basal cell carcinoma cells.

  2009cited by this paper
• RNA repair restores hemoglobin expression in IVS2–654 thalassemic mice

  2009cited by this paper
• Antisense-mediated exon skipping to correct IL-12Rbeta1 deficiency in T cells.

  2009cited by this paper
• Antisense oligonucleotide therapeutics for iron-sulphur cluster deficiency myopathy.

  2009cited by this paper
• A deep intronic mutation in FGB creates 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

  2009cited by this paper
• Antisense oligonucleotide treatment for a pseudoexon‐generating mutation in the NPC1 gene causing Niemann‐Pick type C disease b

  2009cited by this paper
• Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations

  2009cited by this paper
• Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

  2009cited by this paper
• In vivo correction of a Menkes disease model using antisense oligonucleotides

  2008cited by this paper
• c-MYC antisense phosphosphorodiamidate morpholino oligomer inhibits lung metastasis in a murine tumor model.

  2008cited by this paper
• An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models.

  2008cited by this paper
• Identification of a novel antiapoptotic human protein kinase C delta isoform, PKCdeltaVIII in NT2 cells.

  2008cited by this paper
• Antisense oligonucleotide-induced alternative splicing of the APOB mRNA generates a novel isoform of APOB

  2007cited by this paper
• Local dystrophin restoration with antisense oligonucleotide PRO051.

  2007cited by this paper
• U7 snRNA-mediated correction of aberrant splicing caused by activation of cryptic splice sites

  2007cited by this paper
• Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy.

  2007cited by this paper
• Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides

  2007cited by this paper
• Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA.

  2007cited by this paper
• Analysis of Prostate-Specific Membrane Antigen Splice Variants in LNCap Cells

  2006cited by this paper
• Targeted skipping of a single exon harboring a premature termination codon mutation: implications and potential for gene correction therapy for selective dystrophic epidermolysis bullosa patients.

  2006cited by this paper
• Aberrant splicing in the ocular albinism type 1 gene (OA1/GPR143) is corrected in vitro by morpholino antisense oligonucleotides

  2006cited by this paper
• 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

  2006cited by this paper
• Modification of MyD88 mRNA Splicing and Inhibition of IL-1β Signaling in Cell Culture and in Mice with a 2′-O-Methoxyethyl-Modified Oligonucleotide

  2006cited by this paper
• Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements.

  2004cited by this paper
• Disruption of WT1 gene expression and exon 5 splicing following cytotoxic drug treatment: antisense down-regulation of exon 5 alters target gene expression and inhibits cell survival.

  2004cited by this paper
• Cellular Response to an Antisense-mediated Shift of Bcl-x Pre-mRNA Splicing and Antineoplastic Agents*

  2002cited by this paper
• Correction of Alternative Splicing of Tau in Frontotemporal Dementia and Parkinsonism Linked to Chromosome 17*

  2001cited by this paper
• Peptide nucleic acids are potent modulators of endogenous pre-mRNA splicing of the murine interleukin-5 receptor-alpha chain.

  2001cited by this paper
• Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides*

  1999cited by this paper
• Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.

  1993cited by this paper
• Open Access Research Article Modulation of Mdm2 Pre-mrna Splicing by 9-aminoacridine-pna (peptide Nucleic Acid) Conjugates Targeting Intron-exon Junctions

  year unknowncited by this paper

• Alternative Splicing as a Modulator of the Interferon-Gamma Pathway

  2025cites this paper
• Utility of oligonucleotide in upregulating circular RNA production in a cellular model

  2024cites this paper
• Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

  2024cites this paper
• CADASIL from Bench to Bedside: Disease Models and Novel Therapeutic Approaches

  2021cites this paper
• Alternative Splicing in the Nuclear Receptor Superfamily Redirects Human Metabolic Homeostasis

  2020cites this paper
• Alternative Splicing in the Nuclear Receptor Superfamily Expands Gene Function to Refine Endo-Xenobiotic Metabolism

  2020cites this paper
• Alternative splicing of the vitamin D receptor modulates target gene expression and promotes ligand‐independent functions

  2019cites this paper
• Expected future developments in child neurology

  2019cites this paper
• Mechanisms of Oligonucleotide Actions

  2018cites this paper
• Dysferlin Exon 32 Skipping in Patient Cells.

  2018cites this paper
• Splice-Switching Oligonucleotides

  2018cites this paper
• Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides.

  2018cites this paper
• Drugging Pre-mRNA Splicing

  2017cites this paper
• Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain

  2016cites this paper
• Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides

  2016cites this paper
• Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa.

  2016cites this paper
• Therapeutic NOTCH3 cysteine correction in CADASIL using exon skipping: in vitro proof of concept.

  2016cites this paper
• Enhancing the pharmacokinetic/pharmacodynamic properties of therapeutic nucleotides using lipid nanoparticle systems.

  2015cites this paper
• Antisense oligonucleotide-mediated exon skipping of CHRNA1 pre-mRNA as potential therapy for Congenital Myasthenic Syndromes.

  2015cites this paper
• Gene and splicing therapies for neuromuscular diseases.

  2015cites this paper
• Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies

  2015cites this paper
• Synthesis and Exon-Skipping Activity of Chemically Modified RNAs

  2014cites this paper
• Translational and Regulatory Challenges for Exon Skipping Therapies

  2014cites this paper
• Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

  2014cites this paper
• New Innovations: Therapies for Genetic Conditions

  2014cites this paper
• Nanoparticle Delivery of Antisense Oligonucleotides and Their Application in the Exon Skipping Strategy for Duchenne Muscular Dystrophy

  2014cites this paper
• Antisense-Mediated Exon Skipping: Networking to Meet Opportunities and to Overcome Challenges

  2014cites this paper
• Development of Therapeutic Splice-Switching Oligonucleotides

  2014cites this paper
• Antisense oligonucleotide-mediated modulation of SOST and Rank expression

  2014cites this paper
• Medical Biotechnology: Problems and Prospects in Bangladesh

  2013cites this paper
• Splicing therapy for neuromuscular disease☆

  2013cites this paper
• Nanoparticles to Deliver Antisense Oligonucleotides Aimed at Exon Skipping Therapies

  2013cites this paper
• The Application of Clinical Genetics Dovepress Splicing Modulation Therapy in the Treatment of Genetic Diseases

  year unknowncites this paper