ArticleBlood2023
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Article in Blood, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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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.
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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
10 citing papers in PubMed, 16 citations in OpenAlex.
- Human inborn errors of the phagocyte respiratory burst: Chronic granulomatous disease and beyond.Journal of human immunity · 2026Article
- How I Treat: Chronic granulomatous disease.Journal of human immunity · 2026Review
- The Role of NADPH Oxidase 2 in Leukocytes.Antioxidants (Basel, Switzerland) · 2025Review
- Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease.Molecular therapy. Methods & clinical development · 2024Article
- Inflammatory Complications in Chronic Granulomatous Disease.Journal of clinical medicine · 2024Review
- Myelodysplasia and transgene inactivation in X-CGD-γ retroviral gene therapy: The usual suspects and new players.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers.Journal of clinical immunology · 2023Article
- Machine-guided design of synthetic cell type-specificbioRxiv : the preprint server for biology · 2023Article
- Bioinformatics analysis and prediction of Alzheimer's disease and alcohol dependence based on Ferroptosis-related genes.Frontiers in aging neuroscience · 2023Article
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12 authors at 2 institutions in 1 country.
Funding
Abstract
X-linked chronic granulomatous disease (X-CGD) is a primary immunodeficiency caused by mutations in the CYBB gene, resulting in the inability of phagocytic cells to eliminate infections. To design a lentiviral vector (LV) capable of recapitulating the endogenous regulation and expression of CYBB, a bioinformatics-guided approach was used to elucidate the cognate enhancer elements regulating the native CYBB gene. Using this approach, we analyzed a 600-kilobase topologically associated domain of the CYBB gene and identified endogenous enhancer elements to supplement the CYBB promoter to develop MyeloVec, a physiologically regulated LV for the treatment of X-CGD. When compared with an LV currently in clinical trials for X-CGD, MyeloVec showed improved expression, superior gene transfer to hematopoietic stem and progenitor cells (HSPCs), corrected an X-CGD mouse model leading to complete protection against Burkholderia cepacia infection, and restored healthy donor levels of antimicrobial oxidase activity in neutrophils derived from HSPCs from patients with X-CGD. Our findings validate the bioinformatics-guided design approach and have yielded a novel LV with clinical promise for the treatment of X-CGD.
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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.