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Retricted access

Review article

First published 2009-03

Therapeutic Potential of Splice-Switching Oligonucleotides

John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole

Nucleic Acid Therapeutics

Open access

Review article

First published 2014-07

Development of Therapeutic Splice-Switching Oligonucleotides

Petra Disterer, Adrianna Kryczka, Yuqi Liu [...]

Human Gene Therapy

Open access

Research article

First published 2012-06

Aptamer-Mediated Delivery of Splice-Switching Oligonucleotides to the Nuclei of Cancer Cells

Jonathan W. Kotula, Elizabeth D. Pratico, Xin Ming [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2014-02

Design and Application of Bispecific Splice-Switching Oligonucleotides

Burcu Bestas, Graham Mcclorey, Ulf Tedebark [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2014-04

Alternative Splice Forms of CTLA-4 Induced by Antisense Mediated Splice-Switching Influences Autoimmune Diabetes Susceptibility in NOD Mice

Dan V. Mourich, Shannon K. Oda, Frederick J. Schnell [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2021-06

Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization

Suzan M. Hammond, Olga V. Sergeeva, Pavel A. Melnikov [...]

Nucleic Acid Therapeutics

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

Open access

Research article

First published 2015-04

Peptide Nanoparticle Delivery of Charge-Neutral Splice-Switching Morpholino Oligonucleotides

Peter Järver, Eman M. Zaghloul, Andrey A. Arzumanov [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2024-06

Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria

Ainhoa Martínez-Pizarro, Mar álvarez, Maja Dembic [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2016-12

Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity

Cristina S.J. Rocha, Karin E. Lundin, Mark A. Behlke [...]

Nucleic Acid Therapeutics

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