Evidence map›Paper›PMID 16630946›Full record

ReviewThe Journal of allergy and clinical immunology2006

Gene therapy for immune disorders: good news tempered by bad news.

Jennifer M Puck, Harry L Malech

Open access · bronzeAbstract readHistorical ArticleReview
PubMed Publisher
In one paragraph

Review in The Journal of allergy and clinical immunology, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
2.3field-weighted citation impact, top 12% 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

1 citing paper in PubMed, 17 citations in OpenAlex.

  1. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

2 authors at 2 institutions in 1 country.

Jennifer M PuckGenetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. jpuck@mail.nih.gov
Harry L Malech
National Human Genome Research Institute · USNational Institutes of Health · US

Funding

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
Development and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1M
PERIPHERAL BLOOD PROGENITORS-TARGET FOR GENE TRANSFERZ01AI000644 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 1992 to 2008
$4.3M
Gene Therapy Clinical Trials for X-linked Severe Combined ImmunodeficiencyZIAAI000992 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK · 2009 to 2019
$4.1M
GENE THERAPY FOR IMMUNE DEFICIENCIESZ01AI000645 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI DE RAVIN, SUK · 1992 to 2008
$873k
Gene Therapy Clinical Trials for X-linked Severe Combined ImmunodeficiencyZ01AI000992 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI DE RAVIN, SUK · 2007 to 2008
$388k
6 · The paper itself

Abstract

After a dozen years of human gene therapy trials characterized by minimal gene correction and disappointing clinical impact, the field of gene therapy received some good news in 2000. Infants with X-linked severe combined immunodeficiency who received retroviral gene addition to cells from their bone marrow developed impressive immune reconstitution. During the following 2 years, additional patients were treated and the news was even better-babies receiving gene therapy had sustained T-cell production and in several cases developed better cell function than most patients treated with standard bone marrow transplants. Unfortunately, bad news followed. Three of the patients experienced leukemic T-cell expansions, found to be associated with retroviral insertions into genomic DNA. Where does the field stand today?

Indexed as

Genetic TherapyBone Marrow TransplantationHistory, 20th CenturyHistory, 21st CenturyHumansImmunologic Deficiency SyndromesInfantLeukemiaSevere Combined Immunodeficiency

Identifiers

PMID16630946
OpenAlexW1983993040

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.