Evidence map›Paper›PMID 29766024›Full record

ArticleMolecular therapy. Methods & clinical development2018

Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies.

Chang Li, Nikoletta Psatha, Hongjie Wang, Manvendra Singh, Himanshu Bhusan Samal, Wenli Zhang, Anja Ehrhardt, Zsuzsanna Izsvák, Thalia Papayannopoulou, André Lieber

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed
3.8field-weighted citation impact, top 6% 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

29 citing papers in PubMed, 40 citations in OpenAlex.

  1. bioRxiv : the preprint server for biology · 2026
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  3. Molecular therapy. Methods & clinical development · 2025
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  4. Current Non-Viral-Based Strategies to Manufacture CAR-T Cells.International journal of molecular sciences · 2024
    Review
  5. Article
  6. Auto-expansion ofMolecular therapy. Methods & clinical development · 2024
    Article
  7. Review
  8. Article
  9. Review
  10. Review
  11. Human gene therapy · 2022
    Article
  12. Article
  13. Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  14. Article
  15. Article
  16. Article
  17. In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Article
  18. Safe and EffectiveHuman gene therapy · 2021
    Article
  19. Article
  20. ProphylacticCancer research · 2020
    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

10 authors at 4 institutions in 2 countries.

Chang LiDivision of Medical Genetics, Department of Medicine, University of Washington, Box 357720, Seattle, WA 98195, USA.
Nikoletta PsathaDivision of Hematology Department of Medicine, University of Washington, Seattle, WA 98195, USA.
Hongjie WangDivision of Medical Genetics, Department of Medicine, University of Washington, Box 357720, Seattle, WA 98195, USA.
Manvendra SinghMax-Delbrück-Center for Molecular Medicine, Berlin, 13092 Germany.
Himanshu Bhusan SamalMax-Delbrück-Center for Molecular Medicine, Berlin, 13092 Germany.
Wenli ZhangWitten/Herdecke University, Witten, 58448, Germany.
Anja EhrhardtWitten/Herdecke University, Witten, 58448, Germany.
Zsuzsanna IzsvákMax-Delbrück-Center for Molecular Medicine, Berlin, 13092 Germany.
Thalia PapayannopoulouDivision of Hematology Department of Medicine, University of Washington, Seattle, WA 98195, USA.
André LieberDivision of Medical Genetics, Department of Medicine, University of Washington, Box 357720, Seattle, WA 98195, USA.
Max Delbrück Center · DEUniversity of Washington Medical Center · USUniversity of Washington · USWitten/Herdecke University · DE

Funding

Stem Cell and Transplantation BiologyU54DK106829 · NIDDK · FRED HUTCHINSON CANCER RESEARCH CENTER · PI DEREK L STIREWALT · 2015 to 2026
$9.1M
In Vivo Hematopoietic Stem Cell Gene Therapy of Beta-Thalassemia and Sickle Cell DiseaseR01HL130040 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2016 to 2024
$4.3M
Approach for in vivo gene delivery into hematopoietic stem cells for hemophilia A therapyR01HL141781 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2018 to 2021
$2.3M
GENOME EDITING FOR DEVELOPING A TREATMENT FOR BETA GLOBIN DISORDERSR01DK101328 · NIDDK · UNIVERSITY OF WASHINGTON · PI STAMATOYANNOPOULOS, THALIA · 2014 to 2018
$1.7M
In vivo anti-PD1 gene therapyR21CA204036 · NCI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2017 to 2018
$371k
Hematopoietic stem cell based gene therapy of breast cancerR21CA193077 · NCI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2015 to 2016
$370k
NCI NIH HHS R21 CA193077NCI NIH HHS R21 CA204036NHLBI NIH HHS R01 HL130040NHLBI NIH HHS R01 HL141781NIDDK NIH HHS R01 DK101328NIDDK NIH HHS U54 DK106829
6 · The paper itself

Abstract

We generated an integrating, CD46-targeted, helper-dependent adenovirus HDAd5/35++ vector system for hematopoietic stem cell (HSC) gene therapy. The ∼12-kb transgene cassette included a β-globin locus control region (LCR)/promoter driven human γ-globin gene and an elongation factor alpha-1 (EF1α)-mgmt

Indexed as

beta-thalassemiagamma globingene additionhematopoietic stem cellsintegrationsickle cell diseaseSleeping Beauty transposase

Identifiers

PMID29766024
PMCPMC5948227
OpenAlexW2790339057

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.