Evidence map›Paper›PMID 28344987›Full record

ArticleMolecular therapy. Methods & clinical development2017

Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.

Swati Singh, Iram Khan, Socheath Khim, Brenda Seymour, Karen Sommer, Matthew Wielgosz, Zachary Norgaard, Hans-Peter Kiem, Jennifer Adair, Denny Liggitt and 2 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.9field-weighted citation impact, top 18% 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

10 citing papers in PubMed, 13 citations in OpenAlex.

  1. Review
  2. Article
  3. Review
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Toward Combined Cell and Gene Therapy for Genodermatoses.Cold Spring Harbor perspectives in biology · 2020
    Review
  10. Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.Applied biosafety : journal of the American Biological Safety Association · 2020
    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

12 authors at 3 institutions in 1 country.

Swati SinghCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Iram KhanCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Socheath KhimCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Brenda SeymourCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Karen SommerCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Matthew WielgoszDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Zachary NorgaardClinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Hans-Peter KiemClinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA; Department of Pathology, University of Washington, Seattle, WA 98105, USA.
Jennifer AdairClinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA; Department of Medical Oncology, University of Washington, Seattle, WA 98105, USA.
Denny LiggittDepartment of Comparative Medicine, University of Washington, Seattle, WA 98105, USA.
Arthur NienhuisDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
David J RawlingsCenter for Immunity and Immunotherapies and Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA; Department of Pediatrics, University of Washington, Seattle, WA 98105, USA; Department of Immunology, University of Washington, Seattle, WA 98105, USA.
Fred Hutch Cancer Center · USSt. Jude Children's Research Hospital · USUniversity of Washington · US

Funding

VITAMIN AP30CA023074 · NCI · UNIVERSITY OF ARIZONA · PI Dan Theodorescu · 1985 to 2026
$110.2M
VIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI WEISS, MITCHELL J · 1994 to 2022
$43.6M
Vector ProductionP01AI097100 · NIAID · SEATTLE CHILDREN'S HOSPITAL · PI BEARD, BRIAN C · 2012 to 2016
$11.9M
Lentiviral Gene Therapy for Wiskott-Aldrich SyndromeR01AI071163 · NIAID · SEATTLE CHILDREN'S HOSPITAL · PI RAWLINGS, DAVID J · 2008 to 2012
$3.5M
NCI NIH HHS P30 CA023074NHLBI NIH HHS P01 HL053749NIAID NIH HHS P01 AI097100NIAID NIH HHS R01 AI071163
6 · The paper itself

Abstract

Wiskott-Aldrich syndrome (WAS) is a life-threatening immunodeficiency caused by mutations within the

Indexed as

gene therapyinsulated lentivirusWiskott-Aldrich Syndrome

Identifiers

PMID28344987
PMCPMC5363182
OpenAlexW2565961003

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.