Evidence map›Paper›PMID 40777739›Full record

ArticleMolecular therapy. Nucleic acids2025

Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse.

Anne-Fleur E Schneider, Christa L Tanganyika-de Winter, Silvana M G Jirka, Xuyu Tan, Emily G Thompson, Kristen Ha, Anindita Mitra, Stephanie Garcia, Marleen Luimes, Ryan A Oliver and 2 more

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Article
  3. International journal of biological sciences · 2026
    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

12 authors.

Anne-Fleur E SchneiderDepartment of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.
Christa L Tanganyika-de WinterDepartment of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.
Silvana M G JirkaDepartment of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.
Xuyu TanSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Emily G ThompsonSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Kristen HaSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Anindita MitraSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Stephanie GarciaSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Marleen LuimesDepartment of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.
Ryan A OliverSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Vincent GuerlavaisSarepta Therapeutics, First St 215, Cambridge, MA 02142, USA.
Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Centre, Einthovenweg 20, 2333 ZC Leiden, the Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The severe muscle wasting disorder Duchenne muscular dystrophy (DMD) is characterized by the absence of dystrophin, a protein that is essential for muscle stability. Restoring this protein has therapeutic potential. Antisense oligonucleotides (ASOs), designed to target and skip exons, can restore the reading frame that is disrupted in these patients, enabling the production of partially functional dystrophin. Achieving optimal dystrophin restoration remains challenging due to limited delivery and cellular uptake. Muscle homing peptides conjugated to ASOs are a way to achieve this. Previously, CyPep10 (CP10) has been used to significantly increase exon skipping efficiency for the 2'-O-methyl phosphorothioate chemistry in the

Indexed as

antisense oligonucleotidecell-penetrating peptidecyclic peptideDuchenne muscular dystrophyMT: Delivery Strategiesmuscle uptakePMO

Identifiers

PMID40777739
PMCPMC12329520

What OpenQuestion holds

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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.