Evidence map›Paper›PMID 28826231›Full record

ArticleHuman gene therapy2018

Gene Therapy with an Adeno-Associated Viral Vector Expressing Human Interleukin-2 Alters Immune System Homeostasis in Humanized Mice.

Philip A Durost, Ken-Edwin Aryee, Fatima Manzoor, Roland M Tisch, Christian Mueller, Agata Jurczyk, Leonard D Shultz, Michael A Brehm

Open access · greenAbstract read
In one paragraph

Article in Human gene therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
1.8field-weighted citation impact, top 14% 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

13 citing papers in PubMed, 19 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Human Hepatocyte Transduction with Adeno-Associated Virus Vector.Methods in molecular biology (Clifton, N.J.) · 2022
    Article
  6. Review
  7. Article
  8. Article
  9. Review
  10. Innovations in HIV-1 Vaccine Design.Clinical therapeutics · 2020
    Review
  11. Review
  12. Article
  13. 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

8 authors at 3 institutions in 1 country.

Philip A Durost1 Program in Molecular Medicine, University of Massachusetts Medical School , Worcester, Massachusetts.
Ken-Edwin Aryee1 Program in Molecular Medicine, University of Massachusetts Medical School , Worcester, Massachusetts.
Fatima Manzoor2 Department of Immunology and Microbiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
Roland M Tisch2 Department of Immunology and Microbiology, University of North Carolina at Chapel Hill , Chapel Hill, North Carolina.
Christian Mueller3 Department of Pediatrics and Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
Agata Jurczyk1 Program in Molecular Medicine, University of Massachusetts Medical School , Worcester, Massachusetts.
Leonard D Shultz4 Jackson Laboratory , Bar Harbor, Maine.
Michael A Brehm1 Program in Molecular Medicine, University of Massachusetts Medical School , Worcester, Massachusetts.
University of Massachusetts Chan Medical School · USUniversity of North Carolina at Chapel Hill · USJackson Laboratory · US

Funding

Shared Resource ManagementP30CA034196 · NCI · JACKSON LABORATORY · PI William Carl Skarnes · 1985 to 2026
$61.9M
Novel humanized mouse model developed from cord blood CD34 positive HSC and autologous iPS cell derived thymusR01AI132963 · NIAID · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI BREHM, MICHAEL ALLEN, MAEHR, RENE · 2017 to 2021
$4.4M
Humanized Mouse Avatars for T1DUC4DK104218 · NIDDK · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI BREHM, MICHAEL ALLEN, DALEY, GEORGE Q · 2014 to 2014
$4.1M
Development and Validation of Novel NSG Mouse Models for Human Stem Cell TherapyR24OD018259 · OD · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI BREHM, MICHAEL ALLEN, GREINER, DALE LESLIE · 2014 to 2017
$3.0M
Combinatorial Beta Cell-Specific Cytokine Therapy to Reverse Type I DiabetesR01DK103546 · NIDDK · UNIV OF NORTH CAROLINA CHAPEL HILL · PI TISCH, ROLAND M · 2015 to 2018
$1.5M
NCI NIH HHS P30 CA034196NIAID NIH HHS R01 AI132963NIDDK NIH HHS R01 DK103546NIDDK NIH HHS UC4 DK104218NIH HHS R24 OD018259
6 · The paper itself

Abstract

Recombinant adeno-associated viruses (rAAVs) serve as vectors for in vivo gene delivery in both mice and humans, and have broad applicability for the treatment of genetic diseases. Clinical trials with AAV vectors have demonstrated promise and safety in several human diseases. However, the in vivo validation of novel AAV constructs expressing products that act specifically on human cells and tissues is limited by a paucity of effective translatable models. Humanized mice that are engrafted with human cells, tissues, and immune systems offer strong potential to test the biological effectiveness of AAV vectors on human cells and tissues. Using the BLT (bone marrow, liver, thymus) humanized NOD-scid Il2rg

Indexed as

DependovirusGene ExpressionGenetic TherapyHomeostasisInterleukin-2AnimalsGenetic VectorsHumansMiceMice, Inbred NODMice, SCIDT-Lymphocytes, RegulatoryIL2 protein, humanInterleukin-2AAVcytokinehumanizedNSGTreg

Identifiers

PMID28826231
PMCPMC5865247
OpenAlexW2748538138

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.