Evidence map›Paper›PMID 36092365›Full record

ArticleMolecular therapy. Methods & clinical development2022

Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.

Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez, Gayatri Rao, Jonathan D Schwartz, Ken M Law, Julián Sevilla, Paula Río, Rosa Yáñez and 2 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
1.1field-weighted citation impact, top 20% 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

4 citing papers in PubMed, 1 synthesis or guideline pooled it, 6 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Article
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 at 1 institution in 1 country.

Cristina Mesa-NúñezDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Carlos DamiánDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
María Fernández-GarcíaDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Begoña DíezDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Gayatri RaoRocket Pharmaceuticals, Inc., New York, NY, USA.
Jonathan D SchwartzRocket Pharmaceuticals, Inc., New York, NY, USA.
Ken M LawRocket Pharmaceuticals, Inc., New York, NY, USA.
Julián SevillaCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
Paula RíoDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Rosa YáñezDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Juan A BuerenDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Elena AlmarzaDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS- FJD, UAM), Madrid, Spain.
Centre for Biomedical Network Research on Rare Diseases · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Leukocyte adhesion deficiency type I (LAD-I) is a primary immunodeficiency caused by mutations in the

Indexed as

CD18gene therapyhematopoietic stem and progenitor cellsLAD-Ilentiviral vectorleukocyte adhesion deficiency type Ipersonalized medicinepreclinical studiesprimary immunodeficienciesβ2-integrins

Identifiers

PMID36092365
PMCPMC9418989
OpenAlexW4289316226

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.