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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