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Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy

02 January 25 Articles ، Annemieke Aartsma-Rus ، Anastasia Khvorova ، Masad J. Damha Duchenne muscular dystrophy exon 53 skipping ، Locked nucleic acid (LNA) AONs for DMD ، Antisense oligonucleotide therapy DMD ، RNA analysis challenges in exon skipping ، LNA-FRNA and LNA-2′OMe modifications ، hDMDdel52/mdx mouse model for DMD ، Exon skipping efficiency vs. dystrophin restoration ، AON design optimization for DMD treatment
Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy
This study assesses exon 53 skipping in Duchenne muscular dystrophy (DMD) using locked nucleic acid (LNA)-modified antisense oligonucleotides (AONs) in a humanized DMD mouse model. While certain LNA-AONs (LNA-FRNA, LNA-2′OMe) achieved high exon skipping, actual dystrophin restoration was minimal due to RNA interference issues affecting cDNA synthesis. Optimizing AON designs, RNA analysis methods, and alternative exon targets is crucial for improving therapeutic efficacy in DMD exon skipping therapies.
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Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

28 December 24 Articles ، Annemieke Aartsma-Rus ، Judith van Deutekom Next-generation exon skipping AONs ، Duchenne muscular dystrophy exon 51 therapy ، Advanced antisense oligonucleotides for DMD ، Locked nucleic acids (LNAs) in exon skipping ، Becker muscular dystrophy dystrophin restoration ، Novel exon 51 AON chemical modifications ، Preclinical DMD exon skipping studies ، Capillary Western immunoassay dystrophin levels
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
This study advances next-generation exon 51 skipping antisense oligonucleotides (AONs) for Duchenne muscular dystrophy (DMD) by incorporating novel chemical modifications like 5-methylcytosine and locked nucleic acids (LNAs). Screening 100+ AONs identified an optimized target site, achieving 65-fold higher exon skipping and restoring 30-40% dystrophin levels in preclinical models. These AONs demonstrated improved muscle integrity, enhanced motor function, and a favorable safety profile, marking a major step toward safer, more effective DMD therapies.
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Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

30 September 24 Articles ، Annemieke Aartsma-Rus ، Rob W.J. Collin ، Ype Elgersma Individualized ASO therapy for brain diseases ، Dutch Center for RNA Therapeutics (DCRT) ASO development ، Antisense oligonucleotide treatments for genetic disorders ، ASO therapy for rare neurological and eye diseases ، Personalized RNA-based treatments for patients ، Non-profit ASO drug development initiatives ، Global collaboration in ASO therapies
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
This article explores how the Dutch Center for RNA Therapeutics (DCRT) is advancing individualized antisense oligonucleotide (ASO) therapies for rare brain and eye diseases.
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Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides

15 September 24 Articles ، Annemieke Aartsma-Rus N-of-1 antisense oligonucleotide therapy ، Exon skipping ASO design guidelines ، Personalized genetic therapy with ASOs ، Preclinical efficacy testing of ASOs ، Milasen and patient-specific ASO development ، Splice modulation in rare genetic diseases ، Regulatory challenges in N-of-1 therapies
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
This article outlines standardized guidelines for developing N-of-1 exon skipping antisense oligonucleotides, emphasizing preclinical testing, ASO design, and data sharing.
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Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

15 September 24 Articles ، Annemieke Aartsma-Rus Antisense oligonucleotide design strategies ، Splice modulation in exon skipping therapy ، Optimized AONs for Duchenne muscular dystrophy ، AON efficiency factors in gene therapy ، RNA secondary structure prediction for AONs ، Thermodynamics of antisense oligonucleotides ، Exonic splicing enhancers and Tra2β targeting
Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms
This study presents optimized guidelines for designing antisense oligonucleotides, analyzing their sequence composition, binding energy, and role in exon skipping therapies.
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