Commentary 10.1172/JCI131933

Better living through peptide-conjugated chemistry: next-generation antisense oligonucleotides

Elizabeth M. McNally and Brian D. Leverson

Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

Address correspondence to: Elizabeth M McNally, Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois 60611, USA. Phone: 312.503.5600; Email: elizabeth.mcnally@northwestern.edu.

Find articles by McNally, E. in: JCI | PubMed | Google Scholar

Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.

Address correspondence to: Elizabeth M McNally, Center for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, Illinois 60611, USA. Phone: 312.503.5600; Email: elizabeth.mcnally@northwestern.edu.

Find articles by Leverson, B. in: JCI | PubMed | Google Scholar

First published September 30, 2019 - More info

Published in Volume 129, Issue 11 on November 1, 2019
J Clin Invest. 2019;129(11):4570–4571. https://doi.org/10.1172/JCI131933.
© 2019 American Society for Clinical Investigation
First published September 30, 2019 - Version history

Two different antisense oligonucleotide–based (ASO-based) therapies are currently in clinical use to treat neuromuscular diseases. This success, for Duchenne muscular dystrophy and spinal muscular atrophy, offers hope not only for additional neuromuscular diseases, but also for other disorders that could benefit from RNA-targeted therapies. A major limitation for more widespread application of ASOs relates to relatively poor tissue penetration. In this issue of the JCI, Klein et al. showed that conjugating an ASO with an arginine-rich cell-penetrating peptide, Pip6a, enhanced delivery, resulting in corrective outcome for a mouse model of myotonic dystrophy. Linking ASOs to cell-penetrating peptides, or even other moieties, is an approach currently under development with treatment potential to expand to other disorders.

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