Chapter title |
Use of Glucose–Fructose to Enhance the Exon Skipping Efficacy
|
---|---|
Chapter number | 20 |
Book title |
Exon Skipping and Inclusion Therapies
|
Published in |
Methods in molecular biology, September 2018
|
DOI | 10.1007/978-1-4939-8651-4_20 |
Pubmed ID | |
Book ISBNs |
978-1-4939-8650-7, 978-1-4939-8651-4
|
Authors |
Gang Han, Xianjun Gao, Haifang Yin, Han, Gang, Gao, Xianjun, Yin, Haifang |
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 that 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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