ArticleHuman gene therapy methods2017
Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency.
Article in Human gene therapy methods, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 21 citations in OpenAlex.
- An update on diagnosis and treatments of childhood interstitial lung diseases.Breathe (Sheffield, England) · 2025Review
- The Clinical Approach to Interstitial Lung Disease in Childhood: A Narrative Review Article.Children (Basel, Switzerland) · 2024Review
- A toxicology study ofMolecular therapy. Methods & clinical development · 2024Article
- Modulation of plasmacytoid dendritic cell and CD4Archives of virology · 2024Article
- Pulmonary Alveolar Proteinosis and new therapeutic concepts.Klinische Padiatrie · 2024Article
- Emerging Treatments for Childhood Interstitial Lung Disease.Paediatric drugs · 2024Article
- Alveolar macrophages in pulmonary alveolar proteinosis: origin, function, and therapeutic strategies.Frontiers in immunology · 2023Review
- Improving the Safety of Mesenchymal Stem Cell-BasedMolecules and cells · 2022Article
- Article
- New approaches to moderate CRISPR-Cas9 activity: Addressing issues of cellular uptake and endosomal escape.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo.Gene therapy · 2021Review
- Children's Interstitial and Diffuse Lung Diseases (ChILD) in 2020.Children (Basel, Switzerland) · 2020Review
- Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis.Haematologica · 2020Article
- Long-Term Safety and Efficacy of Gene-Pulmonary Macrophage Transplantation Therapy of PAP in Csf2raMolecular therapy : the journal of the American Society of Gene Therapy · 2019Article
Corrections and comments
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Authors and funding
13 authors at 4 institutions in 2 countries.
Funding
Abstract
Hereditary pulmonary alveolar proteinosis (hPAP) is a rare disorder of pulmonary surfactant accumulation and hypoxemic respiratory failure caused by mutations in CSF2RA (encoding the granulocyte/macrophage colony-stimulating factor [GM-CSF] receptor α-chain [CD116]), which results in reduced GM-CSF-dependent pulmonary surfactant clearance by alveolar macrophages. While no pharmacologic therapy currently exists for hPAP, it was recently demonstrated that endotracheal instillation of wild-type or gene-corrected mononuclear phagocytes (pulmonary macrophage transplantation [PMT]) results in a significant and durable therapeutic efficacy in a validated murine model of hPAP. To facilitate the translation of PMT therapy to human hPAP patients, a self-inactivating (SIN) lentiviral vector was generated expressing a codon-optimized human CSF2RA-cDNA driven from an EF1α short promoter (Lv.EFS.CSF2RA
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