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Retricted access

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

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Research article

First published 2024-10

Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β 654 -Thalassemia Mouse Model

Dan Lu, Xiuli Gong, Xinbing Guo [...]

Human Gene Therapy

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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

Open access

Article commentary

First published 2024

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Eric W Ottesen, Ravindra N Singh

Neuroscience Insights

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Research article

First published 2024-08

Alternative Splicing of RNA Is Excessive in Multiple Sclerosis and Not Linked to Gene Expression Levels: Dysregulation Is Corrected by IFN-β

Anthony T. Reder, Aika Goel, Tzintzuni Garcia [...]

Journal of Interferon & Cytokine Research

Open access

Research article

First published 2022-08

Exon Skipping Through Chimeric Antisense U1 snRNAs to Correct Retinitis Pigmentosa GTPase-Regulator ( RPGR ) Splice Defect

Giuseppina Covello, Gehan H. Ibrahim, Niccolò Bacchi [...]

Nucleic Acid Therapeutics

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Brief report

First published 2016-08

Correction of the Pathogenic Alternative Splicing,Caused by the Common GNB3 c.825C>T Allele,Using a Novel,Antisense Morpholino

Jonathan C.P. Mcglinchey, Hemanth Tummala, Douglas H. Lester

Nucleic Acid Therapeutics

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Research article

First published 2021-05

Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β IVS2-654 -Thalassemic Erythroid Progenitor Cells

Annette d'Arqom, Tiwaporn Nualkaew, Natee Jearawiriyapaisarn [...]

Human Gene Therapy

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Research article

First published 2018-9

Chitosan enhances gene delivery of oligonucleotide complexes with magnetic nanoparticles–cell-penetrating peptide

Moataz Dowaidar, Hani Nasser Abdelhamid, Mattias Hällbrink [...]

Journal of Biomaterials Applications

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Research article

First published 2020-9

Catch RNA Splicing Errors to Envision Novel Correctives

Gail Dutton

Genetic Engineering & Biotechnology News

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