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BibTex format

@article{Griesenbach:2026:10.1016/j.omta.2025.201655,
author = {Griesenbach, U and McLachlan, G and Sinadinos, A and Cheminay, C and Ashour, J and Cox, J and Schwotzer, D and Vyhnal, K and Gupta, A and Chan, M and Meng, C and Pineault, K and Castells, E and Dean, RJ and Viegas, MA and Boyd, AC and Davies, JC and Gill, DR and Hyde, SC and Blanset, D and Alton, EW},
doi = {10.1016/j.omta.2025.201655},
journal = {Mol Ther Adv},
title = {F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.},
url = {http://dx.doi.org/10.1016/j.omta.2025.201655},
volume = {34},
year = {2026}
}
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TY  - JOUR
AB  - We have developed a third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV.F/HN) expressing functional cystic fibrosis transmembrane conductance regulator (CFTR) as a gene therapy for cystic fibrosis (BI 3720931). Here, we assessed transduction efficiency and acute toxicology of the rSIV.F/HN vector expressing an enhanced green fluorescent protein (EGFP) reporter gene in non-human primates (NHPs). Intubated male cynomolgus monkeys received one aerosolized dose of vector (n = 3) or placebo (n = 3). Toxicology was assessed by histopathology, clinical pathology, cytokine levels, and changes in body and organ weight. Transduction efficiency was quantified by EGFP immunohistochemistry in airway epithelial cells and vector-specific mRNA and DNA in the lung 7 days post-dosing. There were no vector-related clinical observations, mortalities, or changes in body or organ weight. Clinical pathology and cytokine analyses were unremarkable. Minimal mixed-cell centriacinar inflammation was observed in 1/3 vector-treated animals. Airway epithelial cell transduction efficiency was 9%-12%. Genomic DNA vector integration was detected in 6.7% of lung epithelial cells. Vector-specific mRNA levels were ∼45× endogenous CFTR mRNA levels in lung epithelium and ∼16× in bronchial brushings. This study extends earlier findings of rSIV.F/HN-based in vivo gene transfer in mice to NHPs, demonstrating transduction efficiency without relevant toxicity.
AU  - Griesenbach,U
AU  - McLachlan,G
AU  - Sinadinos,A
AU  - Cheminay,C
AU  - Ashour,J
AU  - Cox,J
AU  - Schwotzer,D
AU  - Vyhnal,K
AU  - Gupta,A
AU  - Chan,M
AU  - Meng,C
AU  - Pineault,K
AU  - Castells,E
AU  - Dean,RJ
AU  - Viegas,MA
AU  - Boyd,AC
AU  - Davies,JC
AU  - Gill,DR
AU  - Hyde,SC
AU  - Blanset,D
AU  - Alton,EW
DO  - 10.1016/j.omta.2025.201655
PY  - 2026///
TI  - F/HN-pseudotyped lentiviral vector efficiently transduces non-human primate airways with no evidence of relevant toxicity.
T2  - Mol Ther Adv
UR  - http://dx.doi.org/10.1016/j.omta.2025.201655
UR  - https://www.ncbi.nlm.nih.gov/pubmed/42460419
VL  - 34
ER  -
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