ArticleMolecular therapy. Methods & clinical development2022
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Erratum issuedOpen access · goldAbstract read
In one paragraph
Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 37 papers.
0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed
5.0field-weighted citation impact, top 4% of its field
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.
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.
Who cites it
37 citing papers in PubMed, 43 citations in OpenAlex.
- Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies.British journal of haematology · 2026Review
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- Hematopoietic (stem) cells-The elixir of life?FEBS letters · 2026Review
- Culturing Potential: advances in ex vivo cell culture systems for haematopoietic cell-based regenerative therapies.Regenerative therapy · 2025Review
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- Lipid nanoparticle-mediated mRNA delivery to CD34Nature biotechnology · 2025Article
- Gene therapy for inborn errors of immunity: past progress, current status and future directions.Rare disease and orphan drugs journal · 2025Article
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
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- Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- In pursuit of an HIV cure: from stem cell transplants to gene therapies.Frontiers in genome editing · 2025Review
- Article
- Introducing a hemoglobin G-Makassar variant in HSCs by in vivo base editing treats sickle cell disease in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Auto-expansion ofMolecular therapy. Methods & clinical development · 2024Article
- Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.Nature nanotechnology · 2024Article
- A simplified G-CSF-free procedure allows for in vivo HSC gene therapy of sickle cell disease in a mouse model.Blood advances · 2024Article
- CRISPR/Cas9 genome editing of CCR5 combined with C46 HIV-1 fusion inhibitor for cellular resistant to R5 and X4 tropic HIV-1.Scientific reports · 2024Article
- Targeted, safe, and efficient gene delivery to human hematopoietic stem and progenitor cells in vivo using the engineered AVID adenovirus vector platform.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
Corrections and comments
- Erratum issued
Authors and funding
19 authors at 5 institutions in 3 countries.
Chang LiUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Hongjie WangUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Sucheol GilUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Audrey GermondWashington National Primate Research Center, Seattle, WA 98195, USA.
Connie FountainWashington National Primate Research Center, Seattle, WA 98195, USA.
Audrey BaldessariWashington National Primate Research Center, Seattle, WA 98195, USA.
Jiho KimUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Zhinan LiuUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Aphrodite GeorgakopoulouUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Stefan RadtkeStem and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Tamás RaskóAG "Mobile DNA Lab" Max Delbrück Center for Molecular Medicine, 13125 Berlin-Buch, Germany.
Amit PandeStem and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Christina ChiangUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Eli ChinUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
Evangelia YannakiGene and Cell Therapy Center, Hematology Department, George Papanicolaou Hospital, Thessaloniki 57010, Greece.
Zsuzsanna IzsvákAG "Mobile DNA Lab" Max Delbrück Center for Molecular Medicine, 13125 Berlin-Buch, Germany.
Thalia PapayannopoulouUniversity of Washington, Department of Medicine, Division of Hematology, Box 357720, Seattle, WA 98195, USA.
Hans-Peter KiemStem and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
André LieberUniversity of Washington, Department of Medicine, Division of Medical Genetics, Box 357720, Seattle, WA 98195, USA.
University of Washington Medical Center · USFred Hutch Cancer Center · USMax Delbrück Center · DEUniversity of Washington · USG. Papanikolaou General Hospital · GR
Funding
Washington National Primate Research CenterP51OD010425 · OD · UNIVERSITY OF WASHINGTON · PI Mari Ostendorf · 2012 to 2026
$201.8MIn Vivo Hematopoietic Stem Cell Gene Therapy of Beta-Thalassemia and Sickle Cell DiseaseR01HL130040 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2016 to 2024
$4.3MIn vivo HSC gene therapy using a multi-modular HDAd vector for HIV cureR01AI174304 · NIAID · UNIVERSITY OF WASHINGTON · PI HANS-PETER KIEM, ANDRE Michael LIEBER · 2023 to 2026
$4.3MApproach for in vivo gene delivery into hematopoietic stem cells for hemophilia A therapyR01HL141781 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2018 to 2021
$2.3MNHLBI NIH HHS R01 HL130040NHLBI NIH HHS R01 HL141781NIAID NIH HHS R01 AI174304NIH HHS P51 OD010425
Abstract
We tested a new
Indexed as
adenovirus vectorgamma globinhematopoietic stem cellshemoglobinopathiesin vivononhuman primates
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
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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.