Abstract
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.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 4570-4571 |
| Number of pages | 2 |
| Journal | Journal of Clinical Investigation |
| Volume | 129 |
| Issue number | 11 |
| DOIs | |
| State | Published - Nov 1 2019 |
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ASJC Scopus subject areas
- Medicine(all)
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Better living through peptide-conjugated chemistry : Next-generation antisense oligonucleotides. / McNally, Elizabeth M.; Leverson, Brian D.
In: Journal of Clinical Investigation, Vol. 129, No. 11, 01.11.2019, p. 4570-4571.Research output: Contribution to journal › Review article
TY - JOUR
T1 - Better living through peptide-conjugated chemistry
T2 - Next-generation antisense oligonucleotides
AU - McNally, Elizabeth M.
AU - Leverson, Brian D.
PY - 2019/11/1
Y1 - 2019/11/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85074378583&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85074378583&partnerID=8YFLogxK
U2 - 10.1172/JCI131933
DO - 10.1172/JCI131933
M3 - Review article
C2 - 31566581
AN - SCOPUS:85074378583
VL - 129
SP - 4570
EP - 4571
JO - Journal of Clinical Investigation
JF - Journal of Clinical Investigation
SN - 0021-9738
IS - 11
ER -