Publications
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, 2021, ESGCTCollaborative Virtual Congress19–22 October 2021Abstracts, Human Gene Therapy, Vol: 32, Pages: A1-A152, ISSN: 1043-0342
Hickmott JW, Prasertsuk P, Bell RV, et al., 2021, Modifying signal peptides for respiratory gene therapy with secreted proteins, ESGCT Collaborative Virtual Congress, Publisher: MARY ANN LIEBERT, INC, Pages: A39-A40, ISSN: 1043-0342
Hickmott JW, Sinadinos AJ, Sergijenko A, et al., 2021, Single cell tools for measuring mRNA and protein expression for gene therapy, ESGCT Collaborative Virtual Congress, Publisher: MARY ANN LIEBERT, INC, Pages: A39-A39, ISSN: 1043-0342
Faulkner N, Alton EW, Griesenbach U, et al., 2021, Reducing anti-vector humoral immunity through modulation of surface glycoprotein density, ESGCT Collaborative Virtual Congress, Publisher: MARY ANN LIEBERT, INC, Pages: A108-A109, ISSN: 1043-0342
Moiseenko A, Pineault K, Sergijenko A, et al., 2021, Functional characterisation of an engineered next generation lentivirus vector for the treatment of cystic fibrosis, ESGCT Collaborative Virtual Congress, Publisher: MARY ANN LIEBERT, INC, Pages: A39-A39, ISSN: 1043-0342
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Juarez-Molina CI, Lund-Palau H, Meng C, et al., 2021, Lentiviral vector/GM-CSF Gene Therapy for Autoimmune Pulmonary Alveolar Proteinosis, ESGCT Collaborative Virtual Congress, Publisher: MARY ANN LIEBERT, INC, Pages: A38-A38, ISSN: 1043-0342
Sinadinos A, Pineault K, Saleh A, et al., 2021, Computational Tools for Airway-Image and Multidimensional-Data Analysis, Publisher: CELL PRESS, Pages: 257-257, ISSN: 1525-0016
Pineault KM, Meng C, Alton EWFW, et al., 2021, Biosafety Risk Assessment: Biodistribution and Environmental Shedding of Topically Administered Lentiviral Vector to the Murine Airway, Publisher: CELL PRESS, Pages: 259-260, ISSN: 1525-0016
Clarke NK, Sinadinos A, Meng C, et al., 2021, Production of Recombinant Protein after Transplantation of Ex Vivo Transduced Macrophages, Publisher: CELL PRESS, Pages: 378-378, ISSN: 1525-0016
Bell RV, Faulkner N, Alton EWFW, et al., 2021, Assessment of F/HN Pseudotyped Lentiviral Vector Following Intravenous Delivery to Mice, 24th Annual Meeting of the American Society of Gene & Cell Therapy, Publisher: CELL PRESS, Pages: 138-139, ISSN: 1525-0016
Juarez-Molina CI, Lund-Palau H, Meng C, et al., 2021, Gene Therapy for Autoimmune Pulmonary Alveolar Proteinosis, Publisher: CELL PRESS, Pages: 256-257, ISSN: 1525-0016
Sinadinos A, Sergijenko A, Meng C, et al., 2021, Development of Lentiviral Vector Respiratory Tract Dosing Methods in Anticipation of Rodent GLP Toxicology Assessment, Publisher: CELL PRESS, Pages: 220-221, ISSN: 1525-0016
Hickmott JW, Prasertsuk P, Bell RV, et al., 2021, Towards Enhancing Signal Peptides for Respiratory Gene Therapy with Secreted Proteins, American Society of Gene and Cell Therapy, Publisher: CELL PRESS, Pages: 139-139, ISSN: 1525-0016
Sergijenko A, Moiseenko A, Pineault K, et al., 2021, Assessment of the Air-Liquid Interface Culture Model as a Tool to Validate Efficacy of a rSIV.F/HN-CFTR Vector, Publisher: CELL PRESS, Pages: 260-260, ISSN: 1525-0016
MacSweeney R, Reddy K, Davies J, et al., 2021, Trans-epithelial nasal potential difference in patients with, and at risk of acute respiratory distress syndrome, Thorax, Vol: 76, Pages: 1099-1107, ISSN: 0040-6376
Background: Impaired alveolar fluid clearance, determined in part by alveolar sodium transport, is associated with acute respiratory distress syndrome (ARDS). Nasal sodium transport may reflect alveolar transport. The primary objective of this prospective, observational study was to determine if reduced nasal sodium transport, as measured by nasal potential difference (NPD), was predictive of the development of and outcome from ARDS.Methods: NPD was measured in 15 healthy controls and in 88 patients: 40 mechanically ventilated patients defined as ‘at-risk’ for ARDS, 61 mechanically ventilated patients with ARDS (13 who were previously included in the ‘at-risk’ group) and 8 ARDS survivors on the ward.Results: In at-risk subjects, maximum NPD (mNPD) was greater in those who developed ARDS (difference –8.4 mV; 95% CI –13.8 to –3.7; p=0.005) and increased mNPD predicted the development of ARDS before its onset (area under the curve (AUC) 0.75; 95% CI 0.59 to 0.89). In the ARDS group, mNPD was not significantly different for survivors and non-survivors (p=0.076), and mNPD was a modest predictor of death (AUC 0.60; 95% CI 0.45 to 0.75). mNPD was greater in subjects with ARDS (−30.8 mV) than in at-risk subjects (−24.2 mV) and controls (−19.9 mV) (p<0.001). NPD values were not significantly different for survivors and controls (p=0.18).Conclusions: Increased NPD predicts the development of ARDS in at-risk subjects but does not predict mortality. NPD increases before ARDS develops, is greater during ARDS, but is not significantly different for controls and survivors. These results may reflect the upregulated sodium transport necessary for alveolar fluid clearance in ARDS. NPD may be useful as a biomarker of endogenous mechanisms to stimulate sodium transport. Larger studies are now needed to confirm these associations and predictive performance.
Martin I, Kenna D, Morales S, et al., 2021, Variability in bacteriophage and antibiotic sensitivity in serial Pseudomonas aeruginosa isolates from cystic fibrosis airway cultures over 12 months, Mircoorganisms, Vol: 9, ISSN: 2076-2607
Antibiotic treatment for Pseudomonas aeruginosa (Pa) in cystic fibrosis is limited in efficacy and may lead to multi-drug resistance (MDR). Alternatives such as bacteriophages are being explored but well designed, and controlled trials are crucial. The rational selection of patients with bacteriophage susceptible infections is required for both safety and efficacy monitoring. We questioned whether bacteriophage susceptibility profiles were constant or variable over time, variability having been reported with antibiotics. Serial Pa isolates (n = 102) from 24 chronically infected cystic fibrosis (CF) patients over one year were investigated with plaque and antibiotic disc diffusion assays. Variable number tandem repeat (VNTR) analysis identified those patients with >1 isolate. A median (range) of 4 (3–6) isolates/patient were studied. Twenty-one (87.5%) individuals had a single VNTR type; three (12.5%) had two VNTR types at different times. Seventy-five percent of isolates were sensitive to bacteriophage at ≥ 1 concentration; 50% of isolates were antibiotic multidrug resistant. Serial isolates, even when representing a single VNTR type, varied in sensitivity to both bacteriophages and antibiotics. The rates of sensitivity to bacteriophage supports the development of this therapy; however, the variability in response has implications for the selection of patients in future trials which must be on the basis of current, not past, isolate testing.
Coates M, Alton E, Rapeport W, et al., 2021, Pseudomonas aeruginosa induces p38MAP kinase-dependent IL-6 and CXCL8 release from bronchial epithelial cells via a Syk kinase pathway, PLoS One, Vol: 16, ISSN: 1932-6203
Pseudomonas aeruginosa (Pa) infection is a major cause of airway inflammation in immunocompromised and cystic fibrosis (CF) patients. Mitogen-activated protein (MAP) and tyrosine kinases are integral to inflammatory responses and are therefore potential targets for novel anti-inflammatory therapies. We have determined the involvement of specific kinases in Pa-induced inflammation. The effects of kinase inhibitors against p38MAPK, MEK 1/2, JNK 1/2, Syk or c-Src, a combination of a p38MAPK with Syk inhibitor, or a novel narrow spectrum kinase inhibitor (NSKI), were evaluated against the release of the proinflammatory cytokine/chemokine, IL-6 and CXCL8 from BEAS-2B and CFBE41o- epithelial cells by Pa. Effects of a Syk inhibitor against phosphorylation of the MAPKs were also evaluated. IL-6 and CXCL8 release by Pa were significantly inhibited by p38MAPK and Syk inhibitors (p<0.05). Phosphorylation of HSP27, but not ERK or JNK, was significantly inhibited by Syk kinase inhibition. A combination of p38MAPK and Syk inhibitors showed synergy against IL-6 and CXCL8 induction and an NSKI completely inhibited IL-6 and CXCL8 at low concentrations. Pa-induced inflammation is dependent on p38MAPK primarily, and Syk partially, which is upstream of p38MAPK. The NSKI suggests that inhibiting specific combinations of kinases is a potent potential therapy for Pa-induced inflammation.
Sergijenko A, Moiseenko A, Pineault K, et al., 2021, LOW LEVELS OF LENTIVIRUS-MEDIATED CFTR GENE TRANSFER ARE SUFFICIENT TO GENERATE ION TRANSPORT CORRECTION IN AIR-LIQUID INTERFACE CULTURES FROM CYSTIC FIBROSIS PATIENTS, Publisher: BMJ PUBLISHING GROUP, Pages: A42-A42, ISSN: 0040-6376
Alton EWFW, Boyd AC, Davies JC, et al., 2021, TOWARDS A FIRST-IN-HUMAN TRIAL WITH A PSEUDOTYPED LENTIVIRUS, Publisher: BMJ PUBLISHING GROUP, Pages: A42-A42, ISSN: 0040-6376
Sinadinos A, Sergijenko A, Meng C, et al., 2021, DEVELOPMENT OF PROTOCOLS FOR MOUSE GLP-TOXICOLOGY STUDIES, Publisher: BMJ PUBLISHING GROUP, Pages: A32-A32, ISSN: 0040-6376
Sinadinos AJ, Sergijenko A, Saleh AD, et al., 2021, QUANTIFICATION OF MRNA AND PROTEIN FROM SINGLE CELLS FOR CYSTIC FIBROSIS GENE THERAPY, Publisher: BMJ PUBLISHING GROUP, Pages: A50-A50, ISSN: 0040-6376
Sinadinos A, Sergijenko A, Saleh A, et al., 2020, SINGLE-CELL ASSAYS FOR QUANTIFYING MRNA AND PROTEIN DURING CYSTIC FIBROSIS GENE THERAPY TRIALS, North American Cystic Fibrosis Conference, Publisher: WILEY, Pages: S203-S203, ISSN: 8755-6863
Sinadinos A, Sergijenko A, Meng C, et al., 2020, DEVELOPMENT OF PROTOCOLS FOR MOUSE GLP-TOXICOLOGY STUDIES, Publisher: WILEY, Pages: S196-S196, ISSN: 8755-6863
Edmondson C, Westrupp N, Wallenburg J, et al., 2020, MONITORING LUNG FUNCTION OF YOUNG PEOPLE WITH CF AT HOME: IS IT RELIABLE? RESULTS FROM THE CLIMB-CF STUDY, Publisher: WILEY, Pages: S290-S290, ISSN: 8755-6863
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Edmondson C, Westrupp N, Wallenburg J, et al., 2020, WHAT IS FEASIBLE WHEN IT COMES TO MONITORING YOUNG PEOPLE WITH CYSTIC FIBROSIS AT HOME? THE RESULTS OF THE CLIMB-CF STUDY, Publisher: WILEY, Pages: S297-S297, ISSN: 8755-6863
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Alton EW, Boyd A, Davies JC, et al., 2020, TOWARDS A FIRST-IN-HUMAN TRIAL WITH A PSEUDOTYPED LENTIVIRUS, Publisher: WILEY, Pages: S224-S224, ISSN: 8755-6863
Sergijenko A, Moiseenko A, Pineault KM, et al., 2020, LOW LEVELS OF CFTR GENE TRANSFER WITH F/HN PSEUDOTYPED LENTIVIRUS ARE SUFFICIENT TO GENERATE ION TRANSPORT CORRECTION IN AIRWAY CELL CULTURES FROM CYSTIC FIBROSIS PATIENTS, Publisher: WILEY, Pages: S73-S73, ISSN: 8755-6863
Pineault KM, Meng C, Griesenbach U, et al., 2020, BIODISTRIBUTION AND ENVIRONMENTAL SHEDDING OF LENTIVIRAL VECTORS FOLLOWING TOPICAL ADMINISTRATION TO MURINE LUNGS, Publisher: WILEY, Pages: S240-S240, ISSN: 8755-6863
Alton EWFW, Boyd AC, Davies JC, et al., 2020, Gene Therapy for Respiratory Diseases: Progress and a Changing Context, HUMAN GENE THERAPY, Vol: 31, Pages: 911-916, ISSN: 1043-0342
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- Citations: 4
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