Evidence map›Paper›PMID 38022635›Full record

ArticleFrontiers in immunology2023

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying

Maria Carmina Castiello, Martina Di Verniere, Elena Draghici, Elena Fontana, Sara Penna, Lucia Sereni, Alessandra Zecchillo, Denise Minuta, Paolo Uva, Marco Zahn and 9 more

Open access · goldAbstract read
In one paragraph

Article in Frontiers in immunology, 2023. 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
1.3field-weighted citation impact, top 16% 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, 4 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Correction of osteopetrosis in the neonateFrontiers in endocrinology · 2024
    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

19 authors at 8 institutions in 4 countries.

Maria Carmina Castiello *San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Martina Di Verniere *San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Elena DraghiciSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Elena FontanaMilan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
Sara PennaSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Lucia SereniSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Alessandra ZecchilloSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Denise MinutaSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Paolo UvaClinical Bioinformatics, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Marco ZahnProtaGene CGT GmbH, Heidelberg, Germany.
Irene Gil-FarinaProtaGene CGT GmbH, Heidelberg, Germany.
Andrea AnnoniSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Silvia IaiaSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Lisa M Ott de BruinWillem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, Netherlands.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Karin Pike-OverzetDepartment of Immunology, Leiden University Medical Center, Leiden, Netherlands.
Frank J T StaalDepartment of Immunology, Leiden University Medical Center, Leiden, Netherlands.
Anna Villa *San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Valentina CapoSan Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
The San Raffaele Telethon Institute for Gene Therapy · ITInstitute of Genetic and Biomedical Research · ITLeiden University Medical Center · NLIstituto di Ricovero e Cura a Carattere Scientifico San RaffaeleProtagen (Germany) · DEIstituti di Ricovero e Cura a Carattere Scientifico · ITIstituto Giannina Gaslini · ITNational Institute of Allergy and Infectious Diseases · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Introduction: Recombination activating genes ( Methods: In this study, we assessed the efficacy of GT in 2 hypomorphic Rag1 murine models (Rag1 Results and discussion: Starting 6 weeks after transplant, GT-treated mice showed a decrease in proportion of myeloid cells and a concomitant increase of B, T and total white blood cells. However, counts remained lower than in mice transplanted with WT Lin- cells. At euthanasia, we observed a general redistribution of immune subsets in tissues, with the appearance of mature recirculating B cells in the bone marrow. In the thymus, we demonstrated correction of the block at double negative stage, with a modest improvement in the cortical/medullary ratio. Analysis of antigenspecific IgM and IgG serum levels after

Indexed as

Immunologic Deficiency SyndromesSevere Combined ImmunodeficiencyAnimalsB-LymphocytesGenetic TherapyHomeodomain ProteinsHumansImmunoproteinsMiceMutationHomeodomain ProteinsImmunoproteinsautoimmunitygene therapyimmune dysregulationimmunodeficiencyleaky SCIDlentiviral vectorsRAG1 gene

Identifiers

PMID38022635
PMCPMC10679457
OpenAlexW4388646956

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.