Paper · 42947239

Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.

Rovina D and 14 others. Genome Med, 2026.

11 sourcespapers-2026-11-12-0.1.0

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ConductScience Library. Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons. Papers, record 42947239. Release papers-2026-11-12-0.1.0, 12 Nov 2026. https://conductscience.org/library/papers/42947239

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Paper

PubMed's record of the paper, with iCite's citation figures. No abstract is shown.

Title: Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.
Journal: Genome Med
Year: 2026
Publication types: Journal Article
PubMed: https://pubmed.ncbi.nlm.nih.gov/42947239/
DOI: https://doi.org/10.1186/s13073-026-01738-3
PMC: https://pmc.ncbi.nlm.nih.gov/articles/PMC13715161/
The journal: https://conductscience.org/library/journal-index/101475844
Revised in PubMed: 2026-10-30

Source PubMed

Authors15

The authors in the order PubMed's record lists them, with the ORCID iDs it states.

Order	Author	Forename
1	Rovina D	Davide
2	Milasi S	Sofia
3	Di Giuseppe G	Giusy
4	Pioner JM	Josè Manuel
5	Langione M	Marianna
6	Giammarino L	Lucrezia
7	Ferrantini C	Cecilia
8	Chiesa M	Mattia
9	Canonico F	Francesco
10	D'Amario D	Domenico

1 to 10 of 15Source PubMed

See all 15 authors

Topics12

NLM's MeSH indexing of the paper, its major topics first.

MeSH term	Major
Induced Pluripotent Stem Cells	Major topic
Dystrophin	Major topic
Muscular Dystrophy, Duchenne	Major topic
CRISPR-Cas Systems	Major topic
Exons	Major topic
Gene Editing	Major topic
Sequence Deletion	Major topic
Humans
Phenotype
Myocytes, Cardiac
Fibroblasts
Cell Differentiation

Source PubMed

Grants9

Each grant PubMed's record writes, matched to the NIH project NIH's grant and paper links name for this paper, and each of those links the record does not write.

Grant, as the record writes it	Agency	Stated by
GR-2021-12375403	Ministero della Salute	PubMed
GR-2021-12375403	Ministero della Salute	PubMed
GR-2021-12375403	Ministero della Salute	PubMed
2764167	Ministero della Salute	PubMed
2764167	Ministero della Salute	PubMed
20223L2C9N	Ministero dell'Università e della Ricerca	PubMed
GUP19012	Fondazione Telethon	PubMed
GUP19012	Fondazione Telethon	PubMed
JTC2018-046	European Commission ERA-NET on Cardiovascular Diseases	PubMed

Source PubMed

Trials0

The trials whose ClinicalTrials.gov record lists the paper, and the trial numbers the paper's PubMed record names.

None in this release.

Comments and corrections0

PubMed's comments and corrections, NLM's own indexing: retractions first, then expressions of concern, errata and updates, and comments last.

None in this release.

References93

The papers the reference list in PubMed's record names, those in our copy of PubMed, in the list's order.

Order	PMID	Title	Journal	Year	In Papers	Source
1	32,977,524	"Betwixt Mine Eye and Heart a League Is Took": The Progress of Induced Pluripotent Stem-Cell-Based Models of Dystrophin-Associated Cardiomyopathy.	Int J Mol Sci	2020	https://conductscience.org/library/papers/32977524	PubMed
2	39,056,750	Cardiomyopathy in Duchenne Muscular Dystrophy and the Potential for Mitochondrial Therapeutics to Improve Treatment Response.	Cells	2024	https://conductscience.org/library/papers/39056750	PubMed
3	27,141,961	Enrichr: a comprehensive gene set enrichment analysis web server 2016 update.	Nucleic Acids Res	2016	https://conductscience.org/library/papers/27141961	PubMed
4	26,676,145	Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy.	Physiol Rev	2016	https://conductscience.org/library/papers/26676145	PubMed
5	23,287,722	RNA-guided human genome engineering via Cas9.	Science	2013	https://conductscience.org/library/papers/23287722	PubMed
6	18,406,573	Early regional myocardial dysfunction in young patients with Duchenne muscular dystrophy.	J Am Soc Echocardiogr	2008	https://conductscience.org/library/papers/18406573	PubMed
7	33,772,245	Generation, functional analysis and applications of isogenic three-dimensional self-aggregating cardiac microtissues from human pluripotent stem cells.	Nat Protoc	2021	https://conductscience.org/library/papers/33772245	PubMed
8	32,656,189	DMD Pluripotent Stem Cell Derived Cardiac Cells Recapitulate in vitro Human Cardiac Pathophysiology.	Front Bioeng Biotechnol	2020	https://conductscience.org/library/papers/32656189	PubMed
9	27,380,939	Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR.	Genome Biol	2016	https://conductscience.org/library/papers/27380939	PubMed
10	1,913,804	Membrane organization of the dystrophin-glycoprotein complex.	Cell	1991	https://conductscience.org/library/papers/1913804	PubMed

1 to 10 of 93

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Cited by1

The papers in our copy of PubMed whose reference list names this one, newest first.

PMID	Title	Journal	Year	In Papers
42,947,239	Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.	Genome Med	2026	https://conductscience.org/library/papers/42947239

Source PubMed

Genes0

The genes the Gene search release ties to the paper: Swiss-Prot's references first, then PubTator's text mining.

None in this release.

Drugs0

The drugs whose trials, in the Pipelines release, list the paper.

None in this release.

Data0

The data bank entries PubMed's record names. Trial numbers are under Trials.

None in this release.

Data dates

The date of each source this release read.

PubMed: 2026-11-06; PubMed
iCite: 2026-11-05; iCite
PMC's ID file: 2026-11-05; PMC's ID file
NIH RePORTER's grant and paper links: 2026-11-01; NIH RePORTER's grant and paper links
Trials: 2026-11-12
Gene search: 2026-11-11
Pipelines: 2026-09-18

Sources 11

ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
HGNCCC0
iCiteCC BY 4.0
NCBI GenePublic domain (US government)
NIH RePORTER's grant and paper linksPublic domain
Open TargetsCC0 1.0
PMC's ID filePublic domain (US government)
PubMedNLM's terms
PubTator 3Public domain
UniProtCC BY 4.0

Each source's terms

ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
HGNCCC0: https://www.genenames.org/about/license/
iCiteCC BY 4.0
NCBI GeneNone named for the file. NCBI says information created by or for the US government on its site is in the public domain, and asks that NLM be acknowledged: https://www.ncbi.nlm.nih.gov/home/about/policies/
NIH RePORTER's grant and paper linksPublic domain (NIH RePORTER)
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PMC's ID fileNone named for the file. NCBI says information created by or for the US government on its site is in the public domain, and asks that NLM be acknowledged: https://www.ncbi.nlm.nih.gov/home/about/policies/
PubMedNLM's terms
PubTator 3Public domain
UniProtCC BY 4.0. UniProt's licence file: "All databases and documents in the UniProt FTP directory may be copied and redistributed freely, without advance permission, provided that this copyright statement is reproduced with each copy." UniProt asks that users cite its latest paper: The UniProt Consortium, UniProt: the Universal Protein Knowledgebase in 2025, Nucleic Acids Research 53:D609-D617, https://doi.org/10.1093/nar/gkae1010. Read on 9 Nov 2026 at https://www.uniprot.org/help/license

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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