ArticleMolecular therapy : the journal of the American Society of Gene Therapy2021
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
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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.
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Who cites it
14 citing papers in PubMed, 23 citations in OpenAlex.
- Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy.Cell reports. Medicine · 2023Trial
- Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Chronic Granulomatous Disease (CGD): Commonly Associated Pathogens, Diagnosis and Treatment.Microorganisms · 2023Review
- Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.Seminars in immunology · 2023Review
- The Arrival of Gene Therapy for Patients with Hemophilia A.International journal of molecular sciences · 2022Review
- Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis.Molecular therapy. Methods & clinical development · 2022Article
- Evaluation of cfDNA as an early detection assay for dense tissue breast cancer.Scientific reports · 2022Article
- Development and clinical translation ofComputational and structural biotechnology journal · 2022Review
- Gene Editing for Inherited Red Blood Cell Diseases.Frontiers in physiology · 2022Review
- Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47Human gene therapy · 2021Article
- Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency.Blood advances · 2021Article
- Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- The Promise and the Hope of Gene Therapy.Frontiers in genome editing · 2021Review
- Gene Therapies for Primary Immune Deficiencies.Frontiers in immunology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vectors (LVs) has emerged as a promising therapeutic option for CGD patients. We performed non-clinical Good Laboratory Practice (GLP) and laboratory-grade studies to assess the safety and genotoxicity of LV targeting myeloid-specific Gp91
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Registered trials
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.