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Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

18 Apr 2024

Abstract excerpt

Although around 6% of the world’s population is affected by rare diseases, only a small number of disease-modifying therapies are available. In recent years, antisense oligonucleotides (ASOs) have emerged as one option for the development of therapeutics for orphan diseases. In particular, ASOs can be utilized for individualized genetic treatments, addressing patients with a known disease-causing genetic variant,...

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PMID: 40225926
DOI: 10.1155/2024/9920230

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