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 DMD ، ExonSkipping ، AntisenseTherapy ، حذف_اگزون ، DystrophinRestoration ، بازسازی_دیستروفین ، LNA ، PreclinicalStudy ، دیستروفی_دوشن ، الیگونوکلئوتید
Challenges of Assessing Exon 53 Skipping of the HumanDMD Transcript with Locked Nucleic Acid-ModifiedAntisense Oligonucleotides in a Mouse Modelfor Duchenne Muscular Dystrophy
  Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, leading to muscle degeneration and early death. Antisense oligonucleotides (AONs) are promising therapeutic agents designed to skip faulty exons in the dystrophin gene and restore functional protein.   This study evaluated the efficiency of AONs targeting exon 53 of the dystrophin gene in a mouse model, using chemically modified AONs to enhance skipping …
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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 DuchenneMuscularDystrophy ، ExonSkipping ، دیستروفی_عضلانی_دوشن ، antisenseoligonucleotide ، پرش_اگزون ، AntisenseOligonucleotide ، DystrophinRestoration ، NextGenerationTherapies ، PreclinicalResearch ، الیگونوکلئوتیدآنتی_سنس ، بازگردانی_دیستروفین ، تحقیقات_پیش_بالینی
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
Antisense oligonucleotides (AONs) have emerged as a promising therapy for Duchenne Muscular Dystrophy (DMD), a severe neuromuscular disease caused by dystrophin deficiency. Early AON drugs like drisapersen and eteplirsen achieved limited success, leading researchers to develop more efficient next-generation AONs. This study focused on refining AONs for exon 51 skipping through advanced chemical modifications and identifying optimal target sites to enhance efficacy and safety.   Researchers screened over 100 AONs …
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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 AntisenseOligonucleotides ، NOf1Therapy ، IndividualizedTreatment ، RareDiseases ، RNAtherapeutics ، Nof1Therapy ، بیماری‌های_نادر ، درمان_فردی ، الیگونوکلئوتید_ضدحس ، درمان_تک‌فردی
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
Personal take on this article: This paper discusses the work of the Dutch Center for RNA Therapeutics (DCRT) in developing individualized antisense oligonucleotides (ASOs) for patients with rare brain or eye diseases. ASOs are short pieces of modified DNA that can adjust gene expression and have the potential to treat genetic disorders. However, many rare diseases are specific to individual patients, meaning pharmaceutical companies often overlook these cases due to …
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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 GeneTherapy ، ExonSkipping ، ASO ، PersonalizedMedicine ، NOf1Therapy ، آنتی‌سنس_الیگو‌نوکلئوتید ، پریدن_اکسون ، درمان_ژنی ، پزشکی_شخصی_سازی_شده ، درمان_برای_یک_فرد
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
    Personal take on this article: This paper outlines consensus guidelines for the design and testing of antisense oligonucleotides (ASOs) specifically tailored to individual patients, known as N-of-1 therapies. ASOs are short strands of synthetic DNA that can modify gene expression by altering pre-mRNA splicing, offering potential treatments for various genetic diseases. The paper highlights the importance of designing ASOs that skip certain exons to restore protein function, which …
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Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

15 September 24 Articles ، Annemieke Aartsma-Rus DuchenneMuscularDystrophy ، GeneTherapy ، ExonSkipping ، AntisenseOligonucleotides ، دیستروفی_عضلانی_دوشن ، SpliceModulation ، ESEMotifs ، درمان_ژن
Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms
    Personal take on this article: The paper provides a comprehensive set of guidelines for designing antisense oligonucleotides (AONs) with a focus on splice modulation. AONs are tools used to interfere with gene expression by targeting specific mRNA sequences, either to degrade them, block translation, or modify splicing. This study particularly focuses on the latter, using AONs to induce exon skipping in the dystrophin gene, which has therapeutic potential …
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