Citation:
Methods Mol Biol. 2018;1828:327-342. doi: 10.1007/978-1-4939-8651-4_20
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.
Epub:
Not Epub
Link to Publication:
https://link.springer.com/protocol/10.1007%2F978-1-4939-8651-4_20
Organism or Cell Type:
mice mdx
Delivery Method:
injection