ArticleMolecular therapy : the journal of the American Society of Gene Therapy2019
Long-Term Safety and Efficacy of Gene-Pulmonary Macrophage Transplantation Therapy of PAP in Csf2ra
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed, 32 citations in OpenAlex.
- The impact of macrophage metabolism on idiopathic pulmonary fibrosis development and treatment prospectives.Frontiers in immunology · 2026Review
- A generalized protocol for the induction of M2-like macrophages from mouse and rat bone marrow mononuclear cells.Biology methods & protocols · 2025Article
- Rare genetic interstitial lung diseases: a pictorial essay.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Pathogenesis-driven treatment of primary pulmonary alveolar proteinosis.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- A toxicology study ofMolecular therapy. Methods & clinical development · 2024Article
- Article
- Pulmonary Alveolar Proteinosis and new therapeutic concepts.Klinische Padiatrie · 2024Article
- Ten-year experience of whole lung lavage in pediatric Pulmonary Alveolar Proteinosis.Klinische Padiatrie · 2024Article
- Alveolar macrophages in tissue homeostasis, inflammation, and infection: evolving concepts of therapeutic targeting.The Journal of clinical investigation · 2023Review
- Alveolar macrophages in pulmonary alveolar proteinosis: origin, function, and therapeutic strategies.Frontiers in immunology · 2023Review
- Progress in Respiratory Gene Therapy.Human gene therapy · 2022Review
- Autoimmune Pulmonary Alveolar Proteinosis.American journal of respiratory and critical care medicine · 2022Article
- A murine model of hereditary pulmonary alveolar proteinosis caused by homozygousAmerican journal of physiology. Lung cellular and molecular physiology · 2022Article
- Role of GM-CSF in regulating metabolism and mitochondrial functions critical to macrophage proliferation.Mitochondrion · 2022Article
- Transcriptome analysis of molecular mechanisms underlying facial nerve injury repair in rats.Neural regeneration research · 2021Article
- Beyond "Big Eaters": The Versatile Role of Alveolar Macrophages in Health and Disease.International journal of molecular sciences · 2021Review
- Alveolar proteinosis of genetic origins.European respiratory review : an official journal of the European Respiratory Society · 2020Article
- Integrative Identification of Hub Genes Associated With Immune Cells in Atrial Fibrillation Using Weighted Gene Correlation Network Analysis.Frontiers in cardiovascular medicine · 2020Article
Corrections and comments
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Authors and funding
14 authors at 3 institutions in 3 countries.
Funding
Abstract
Hereditary pulmonary alveolar proteinosis (PAP) is a genetic lung disease characterized by surfactant accumulation and respiratory failure arising from disruption of GM-CSF signaling. While mutations in either CSF2RA or CSF2RB (encoding GM-CSF receptor α or β chains, respectively) can cause PAP, α chain mutations are responsible in most patients. Pulmonary macrophage transplantation (PMT) is a promising new cell therapy in development; however, no studies have evaluated this approach for hereditary PAP (hPAP) caused by Csf2ra mutations. Here, we report on the preclinical safety, tolerability, and efficacy of lentiviral-vector (LV)-mediated Csf2ra expression in macrophages and PMT of gene-corrected macrophages (gene-PMT therapy) in Csf2ra gene-ablated (Csf2ra
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Registered trials
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