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Use of Glucose/Fructose to Enhance the Exon Skipping Efficacy
1School of Medical Technology & Department of Cell Biology, Tianjin Medical University, Tianjin, China.
Abstract:
Exon skipping antisense oligonucleotides (AOs) are promising treatments for muscle-related genetic ailments, including Duchenne muscular dystrophy (DMD), but clinical translation is unfortunately hampered by insufficient systemic delivery. Here, we describe how one can employ a glucose/fructose injection mixture to improve muscle uptake and functional outcomes of DMD AOs in energy-deficient peripheral muscles of mdx mice. The potentiating effect of glucose/fructose on AOs in energy-deficient muscles offers a simple and economical method for enhancing AO potency, reducing screening costs for researchers and accelerating the translation of nucleic acid-based therapeutics in DMD and other muscular dystrophies.
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