Evidence map›Paper›PMID 41028684›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2026

Utilization of Induced Pluripotent Stem Cell-Derived Neurons to Investigate the Splice-Modification Efficacy of Splice-Switching Drug Candidates.

Yukiko Ueta, Hiroaki Ohara, Tomonari Awaya, Masatoshi Hagiwara

Abstract read
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In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

4 authors.

Yukiko UetaDepartment of Drug Discovery Medicine, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Hiroaki OharaDepartment of Drug Discovery Medicine, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Tomonari AwayaCenter for Anatomical Studies, Graduate School of Medicine, Kyoto University, Kyoto, Japan. awaya@kuhp.kyoto-u.ac.jp.
Masatoshi HagiwaraDepartment of Drug Discovery Medicine, Graduate School of Medicine, Kyoto University, Kyoto, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Modification of RNA splicing represents a promising therapeutic strategy for treating genetic diseases. Several splice-switching drugs have already been approved, notably for spinal muscular atrophy and specific genotypes of Duchenne muscular dystrophy. While peripheral blood mononuclear cells and fibroblasts are commonly used to evaluate the efficacy of these drug candidates in patient-derived samples, such approaches may lead to misleading conclusions due to the cell type-specific nature of RNA splicing.Induced pluripotent stem cells (iPSCs), which can be differentiated into various somatic cell types, offer a valuable tool to overcome this limitation. Once efficient differentiation protocols are established, iPSCs can give rise to neurons, retinal cells, cardiomyocytes, and other hard-to-obtain cell types. In this chapter, we describe a protocol for evaluating the splice-modification efficacy of drug candidates in iPSC-derived neurons, using the removal of a poison exon in SCN1A gene, associated with Dravet syndrome as a model.

Indexed as

Drug DiscoveryNeuronsRNA SplicingCell DifferentiationCulture MediaEpilepsies, MyoclonicExonsHumansInduced Pluripotent Stem CellsNAV1.1 Voltage-Gated Sodium ChannelRNACulture MediaNAV1.1 Voltage-Gated Sodium ChannelRNASCN1A protein, humanAntisense oligonucleotidesFunctional validation of splicing correctionInduced pluripotent stem cells (iPSCs)RNA splicingSmall-molecule compoundsSplice-switching oligonucleotides (SSOs)

Identifiers

PMID41028684

What OpenQuestion holds

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Registered trials

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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.