Evidence map›Paper›PMID 38010230›Full record

ArticleNucleic acid therapeutics2023

Challenges of Assessing Exon 53 Skipping of the Human

Sarah Engelbeen, Daniel O'Reilly, Davy Van De Vijver, Ingrid Verhaart, Maaike van Putten, Vignesh Hariharan, Matthew Hassler, Anastasia Khvorova, Masad J Damha, Annemieke Aartsma-Rus

Open access · hybridAbstract read
In one paragraph

Article in Nucleic acid therapeutics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.7field-weighted citation impact, top 15% of its field
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

9 citing papers in PubMed, 11 citations in OpenAlex.

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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

10 authors at 3 institutions in 3 countries.

Sarah EngelbeenDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0002-5358-8691
Daniel O'ReillyUniversity of Massachusetts Chan Medical School, RNA Therapeutics Institute, Worcester, Massachusetts, USA.
Davy Van De VijverDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Ingrid VerhaartDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0001-7348-7989
Maaike van PuttenDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Vignesh HariharanUniversity of Massachusetts Chan Medical School, RNA Therapeutics Institute, Worcester, Massachusetts, USA.
Matthew HasslerUniversity of Massachusetts Chan Medical School, RNA Therapeutics Institute, Worcester, Massachusetts, USA.
Anastasia KhvorovaUniversity of Massachusetts Chan Medical School, RNA Therapeutics Institute, Worcester, Massachusetts, USA.
Masad J DamhaDepartment of Chemistry, McGill University, Montreal, Canada.
Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0003-1565-654X
Leiden University Medical Center · NLUniversity of Massachusetts Chan Medical School · USMcGill University · CA

Funding

Mid-Scale RNA Synthesis, Purification and Quality Control SystemS10OD020012 · OD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI KHVOROVA, ANASTASIA · 2015 to 2015
$563k
NIH HHS S10 OD020012US Department of Defense W81XWH-22-1-0744
6 · The paper itself

Abstract

Antisense oligonucleotide (AON)-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression by reframing the disrupted open reading frame of the

Indexed as

DystrophinMuscular Dystrophy, DuchenneAnimalsExonsGenetic TherapyHumansMaleMiceMice, Inbred mdxOligonucleotidesOligonucleotides, AntisenseRNADystrophinlocked nucleic acidOligonucleotidesOligonucleotides, AntisenseRNAefficacyexon skippingLNAlocked nucleic acidpreclinical studies

Identifiers

PMID38010230
PMCPMC10698779
OpenAlexW4389047030

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.