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has been cited by the following article:

• TITLE: Rare Diseases and Antisense Oligonucleotides: A Mirage or Miracle

  AUTHORS: Harika Vaddadi, Terry Oroszi

  KEYWORDS: Antisense Oligonucleotides, Rare Diseases, Gene Therapy, Off-Target Effects, Duchenne Muscular Dystrophy, Timothy Syndrome

  JOURNAL NAME: Journal of Biosciences and Medicines, Vol.12 No.12, December 2, 2024

  ABSTRACT: Gene therapy and antisense oligonucleotides (ASOs) are promising approaches to treating rare diseases by targeting specific genes. However, ASOs can have off-target effects that need careful consideration during development. Researchers can add moieties like peptide nucleic acid or methoxyethyl-modified ribose sugars to enhance specificity and reduce toxicity. Current research suggests that challenges such as nonspecific action, interference at various stages, adverse reactions, and nuclease degradation may soon be manageable with advanced technologies. ASOs show particular promise in treating rare conditions like Duchenne Muscular Dystrophy (DMD) and Timothy syndrome. Stereopure ASOs with repeated left-right patterns offer increased potency and half-life due to their resistance to nuclease activity and improved cellular uptake. This review explores how technological advancements can enhance the use of ASOs to manage various rare disease conditions effectively. Despite challenges in development and application, ASO therapy holds the potential to become a viable treatment option for a wide range of rare diseases. Advances in technology offer the possibility of increasing specificity and reducing toxicity, making ASO therapy a more effective and safe treatment option for patients with rare diseases.