Evidence map›Paper›PMID 29479836›Full record

ArticleAngewandte Chemie (International ed. in English)2018

Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides.

Justin M Wolfe, Colin M Fadzen, Rebecca L Holden, Monica Yao, Gunnar J Hanson, Bradley L Pentelute

Abstract read
In one paragraph

Article in Angewandte Chemie (International ed. in English), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

34 citing papers in PubMed.

  1. The Versatile Applications of Antisense Oligonucleotides in Modern Medicine.International journal of molecular sciences · 2026
    Review
  2. Article
  3. Bismuth Bicycles.Journal of peptide science : an official publication of the European Peptide Society · 2026
    Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Dynamic carriers for therapeutic RNA delivery.Proceedings of the National Academy of Sciences of the United States of America · 2024
    Article
  10. Article
  11. Review
  12. Article
  13. Article
  14. Article
  15. Review
  16. A Pt(IV)-conjugated brain penetrant macrocyclic peptide shows pre-clinical efficacy in glioblastoma.Journal of controlled release : official journal of the Controlled Release Society · 2022
    Article
  17. Article
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Justin M WolfeDepartment of Chemistry, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, MA, 02139, USA.ORCID 0000-0002-1003-6045
Colin M FadzenDepartment of Chemistry, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, MA, 02139, USA.ORCID 0000-0002-4139-9578
Rebecca L HoldenDepartment of Chemistry, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, MA, 02139, USA.
Monica YaoResearch Chemistry, Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA, 02142, USA.
Gunnar J HansonResearch Chemistry, Sarepta Therapeutics, Inc., 215 First Street, Cambridge, MA, 02142, USA.
Bradley L PenteluteDepartment of Chemistry, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, MA, 02139, USA.ORCID 0000-0002-7242-801X

Funding

Chemically Modified Peptide Agents for Next-Generation Conjugate Therapies to Treat Duchenne Muscular DystrophyF30HD093358 · NICHD · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI FADZEN, COLIN MACLAINE · 2017 to 2019
$142k
NICHD NIH HHS F30 HD093358
6 · The paper itself

Abstract

Exon-skipping antisense oligonucleotides are effective treatments for genetic diseases, yet exon-skipping activity requires that these macromolecules reach the nucleus. While cell-penetrating peptides can improve delivery, proteolytic instability often limits efficacy. It is hypothesized that the bicyclization of arginine-rich peptides would improve their stability and their ability to deliver oligonucleotides into the nucleus. Two methods were introduced for the synthesis of arginine-rich bicyclic peptides using cysteine perfluoroarylation chemistry. Then, the bicyclic peptides were covalently linked to a phosphorodiamidate morpholino oligonucleotide (PMO) and assayed for exon skipping activity. The perfluoroaryl cyclic and bicyclic peptides improved PMO activity roughly 14-fold over the unconjugated PMO. The bicyclic peptides exhibited increased proteolytic stability relative to the monocycle, demonstrating that perfluoroaryl bicyclic peptides are potent and stable delivery agents.

Indexed as

Drug Delivery SystemsBridged Bicyclo CompoundsCell-Penetrating PeptidesDrug CarriersFluorocarbonsHeLa CellsHumansMolecular StructureOligonucleotides, AntisenseBridged Bicyclo CompoundsCell-Penetrating PeptidesDrug CarriersFluorocarbonsOligonucleotides, Antisenseantisense agentsbicyclizationcell-penetrating peptidescysteine arylationpeptides

Identifiers

PMID29479836
PMCPMC6248909

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.