Sage Journals: Discover world-class research

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Meeting report

First published 2025-12

Risdiplam: A Small Molecule mRNA Splice Modifier Approved to Treat SMA Disease

Lutz Müller, Björn Jacobsen, Alexander Nürnberg

Toxicologic Pathology

Open access

Research article

First published 2022-06

2′- O -( N -(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity

Dmytro Honcharenko, Cristina S.J. Rocha, Karin E. Lundin [...]

Nucleic Acid Therapeutics

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Review article

First published 2004-03

Modification of Alternative Splicing by Antisense Therapeutics

Ryszard Kole, Marla Vacek, Tiffany Williams

Nucleic Acid Therapeutics

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Research article

First published 2009-03

Noncovalent Peptide-Mediated Delivery of Chemically Modified Steric Block Oligonucleotides Promotes Splice Correction: Quantitative Analysis of Uptake and Biological Effect

Sandra D. Laufer, Anna Lena Recke, Sandra Veldhoen [...]

Nucleic Acid Therapeutics

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Research article

First published 2013-01

A Gene Therapeutic Approach to Correct Splice Defects with Modified U1 and U6 snRNPs

Fabian Schmid, Thomas Hiller, Germaine Korner [...]

Human Gene Therapy

Open access

Research article

First published 2017-4

Effect of histone modifications on hMLH1 alternative splicing in gastric cancer

Jin-Xuan Zhao, Xiao-Wei Li, Bing-Yu Shi [...]

Tumor Biology

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Research article

First published 2019-04

A SMN2 Splicing Modifier Rescues the Disease Phenotypes in an In Vitro Human Spinal Muscular Atrophy Model

Ye Seul Son, Kwangman Choi, Hana Lee [...]

Stem Cells and Development

Free access

Research article

First published 2019-1

Development of a Screening Platform to Identify Small Molecules That Modify ELP1 Pre-mRNA Splicing in Familial Dysautonomia

Monica Salani, Fabio Urbina, Anthony Brenner [...]

Journal of Biomolecular Screening

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Review article

First published 2009-03

Therapeutic Potential of Splice-Switching Oligonucleotides

John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole

Nucleic Acid Therapeutics

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Abstract

First published 2006-1

Erythropoietin Supplementation Alters Cerebral Covalent Histone Modification and Glucocorticoid Receptor Splice Variant Messenger Ribonucleic Acid Expression in a Gender-Specific Manner

D.J. Braunreiter, R.J. McPherson, R.A. McKnight [...]

Journal of Investigative Medicine

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