ArticleAmerican journal of respiratory and critical care medicine2012
Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.
Article in American journal of respiratory and critical care medicine, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers.
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Who cites it
58 citing papers in PubMed, 101 citations in OpenAlex.
- Article
- Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice.Gene therapy · 2026Article
- Using the nose as a factory to secrete proteins into the lungs or circulation.Molecular therapy. Advances · 2026Article
- Inhalable gene and RNA therapy for cystic fibrosis: perspectives and progress in clinical development.Nanomedicine (London, England) · 2026Review
- F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.Molecular therapy. Advances · 2026Article
- Optimized Lentiviral Vector Production Using Dual-Pseudotyping with VSV-G and Sendai Virus HN Glycoproteins for Enhanced Gene Delivery in Diverse Cell Types.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy.The European respiratory journal · 2025Article
- The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium.American journal of respiratory and critical care medicine · 2024Article
- Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.American journal of respiratory and critical care medicine · 2024Review
- Article
- Lentiviral expression of wild-type LAMA3A restores cell adhesion in airway basal cells from children with epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Advances in Genetic Reprogramming: Prospects from Developmental Biology to Regenerative Medicine.Current medicinal chemistry · 2024Review
- The Dual Role of the Airway Epithelium in Asthma: Active Barrier and Regulator of Inflammation.Cells · 2023Review
- Repeat or single-dose lentiviral vector administration to mouse lungs? It's all about the timing.Gene therapy · 2023Article
- Effective viral-mediated lung gene therapy: is airway surface preparation necessary?Gene therapy · 2023Review
- Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2023Article
- Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell.Human gene therapy · 2022Review
- Sendai F/HN pseudotyped lentiviral vector transduces human ciliated and non-ciliated airway cells using α 2,3 sialylated receptors.Molecular therapy. Methods & clinical development · 2022Article
- Progress in Respiratory Gene Therapy.Human gene therapy · 2022Review
- Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression.Molecular therapy. Methods & clinical development · 2022Article
Corrections and comments
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Authors and funding
19 authors at 5 institutions in 2 countries.
Funding
Abstract
rationaleOngoing efforts to improve pulmonary gene transfer thereby enabling gene therapy for the treatment of lung diseases, such as cystic fibrosis (CF), has led to the assessment of a lentiviral vector (simian immunodeficiency virus [SIV]) pseudotyped with the Sendai virus envelope proteins F and HN.
objectivesTo place this vector onto a translational pathway to the clinic by addressing some key milestones that have to be achieved.
methodsF/HN-SIV transduction efficiency, duration of expression, and toxicity were assessed in mice. In addition, F/HN-SIV was assessed in differentiated human air-liquid interface cultures, primary human nasal epithelial cells, and human and sheep lung slices. MEASUREMENTS AND MAIN
resultsA single dose produces lung expression for the lifetime of the mouse (~2 yr). Only brief contact time is needed to achieve transduction. Repeated daily administration leads to a dose-related increase in gene expression. Repeated monthly administration to mouse lower airways is feasible without loss of gene expression. There is no evidence of chronic toxicity during a 2-year study period. F/HN-SIV leads to persistent gene expression in human differentiated airway cultures and human lung slices and transduces freshly obtained primary human airway epithelial cells.
conclusionsThe data support F/HN-pseudotyped SIV as a promising vector for pulmonary gene therapy for several diseases including CF. We are now undertaking the necessary refinements to progress this vector into clinical trials.
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