Evidence map›Paper›PMID 42564579›Full record

ArticleMolecular therapy. Advances2026

The potential of gene therapy to rescue telomeric and extratelomeric defects in X-linked dyskeratosis congenita.

Diego Llorente-Arroyo, María Luz Lozano, Susana Navarro, Leandro Sastre, Rosario Perona, Juan Antonio Bueren, Guillermo Guenechea, Nestor Wenceslao Meza

Abstract read
In one paragraph

Article in Molecular therapy. Advances, 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

8 authors.

Diego Llorente-ArroyoDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.
María Luz LozanoDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.
Susana NavarroDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.
Leandro SastreInstituto de Investigaciones Biomédicas "Alberto Sols" (CSIC/UAM) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 28029 Madrid, Spain.
Rosario PeronaInstituto de Investigaciones Biomédicas "Alberto Sols" (CSIC/UAM) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 28029 Madrid, Spain.
Juan Antonio BuerenDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.
Guillermo GuenecheaDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.
Nestor Wenceslao MezaDivision of Hematopoietic Innovative Therapies, Biomedical Innovation Unit, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Advanced Therapies Unit - Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), 28040 Madrid, Spain.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Dyskeratosis congenita (DC) is a rare inherited disorder characterized by bone marrow failure, pulmonary fibrosis, and cancer predisposition. X-linked DC (X-DC) is caused by mutations in the

Indexed as

DKC1dyskerinlentiviral gene therapyrRNA maturationtelomere maintenanceX-linked dyskeratosis congenita

Identifiers

PMID42564579
PMCPMC13444285

What Socratic 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 Socratic graph.