Evidence map›Paper›PMID 29746676›Full record

ReviewJournal of the Pediatric Infectious Diseases Society2018

Future of Care for Patients With Chronic Granulomatous Disease: Gene Therapy and Targeted Molecular Medicine.

Michael D Keller, Luigi D Notarangelo, Harry L Malech

Open access · bronzeAbstract readReview
In one paragraph

Review in Journal of the Pediatric Infectious Diseases Society, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
2.1field-weighted citation impact, top 13% 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

11 citing papers in PubMed, 31 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Article
  5. Article
  6. NOX Inhibitors: From Bench to Naxibs to Bedside.Handbook of experimental pharmacology · 2021
    Article
  7. Late diagnosis of chronic granulomatous disease.Clinical and experimental immunology · 2020
    Article
  8. Article
  9. Review
  10. Review
  11. Review
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

3 authors at 2 institutions in 1 country.

Michael D KellerDivision of Allergy and Immunology, Children's National Medical Center, Washington, DC.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Disease, Bethesda, Maryland.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Disease, Bethesda, Maryland.
National Institute of Allergy and Infectious Diseases · USChildren's National · US

Funding

Characterization of the cellular and molecular bases of inborn errors of immunityZIAAI001222 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI NOTARANGELO, LUIGI · 2017 to 2025
$15.2M
Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Basic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5M
Gene Therapy Clinical Trials for Chronic Granulomatous DiseaseZIAAI000991 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI KANG, ELIZABETH · 2009 to 2019
$3.4M
6 · The paper itself

Abstract

Chronic granulomatous disease is a rare and potentially fatal disorder of neutrophil function. Beyond current medical management and hematopoietic stem cell transplantation, new methods of gene therapy that use lentiviral vectors or gene editing might extend curative therapies to patients who lack a suitable transplantation donor while eliminating the risk of graft-versus-host disease. Furthermore, new therapies focused on altering the biology of phagolysosomes might offer novel targeted treatments for inflammatory complications in patients with chronic granulomatous disease.

Indexed as

Genetic TherapyGenetic VectorsGranulomatous Disease, ChronicHumansLentivirusLoss of Function Mutation

Identifiers

PMID29746676
PMCPMC5985732
OpenAlexW2802474374

What OpenQuestion holds

Textmetadata
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.