Publications
271 results found
Allaire NE, Griesenbach U, Kerem B, et al., 2023, Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis., J Cyst Fibros, Vol: 22 Suppl 1, Pages: S39-S44
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF).
Sinadinos A, Bell R, Meng C, et al., 2022, A lentiviral vector intranasal dosing strategy to control local and systemic expression of intracellular and secreted transgenic proteins in vivo, 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Publisher: MARY ANN LIEBERT, INC, Pages: A195-A195, ISSN: 1043-0342
Fiore MJ, Bell RV, Hickmott JW, et al., 2022, Significant transgene repression using the dCas9-KRAB-MeCP2 repressor system, 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Publisher: MARY ANN LIEBERT, INC, Pages: A151-A152, ISSN: 1043-0342
Richardson VR, Clarke NK, Griesenbach U, et al., 2022, Optimising the genetic modification of macrophages using a lentiviral vector for application in cellular therapies, 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Publisher: MARY ANN LIEBERT, INC, Pages: A43-A43, ISSN: 1043-0342
Juarez-Molina C, Meng C, Morgan C, et al., 2022, Non-viral Gene Therapy for Autoimmune Pulmonary Alveolar Proteinosis, 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Publisher: MARY ANN LIEBERT, INC, Pages: A189-A189, ISSN: 1043-0342
Bell RV, Clarke NK, Alton EWFW, et al., 2022, Regulated expression of secreted transgenes by pulmonary transplanted macrophages in mice, 29th Annual Congress of the European-Society-of-Gene-and-Cell-Therapy (ESCGT), Publisher: MARY ANN LIEBERT, INC, Pages: A55-A55, ISSN: 1043-0342
McLachlan G, Alton EWFW, Boyd AC, et al., 2022, Progress in Respiratory Gene Therapy, HUMAN GENE THERAPY, Vol: 33, Pages: 893-912, ISSN: 1043-0342
Alton E, Lund-Palau H, Juarez-Molina C, et al., 2022, Correction of a chronic pulmonary disease through lentiviral vector-mediated protein expression, Molecular Therapy - Methods and Clinical Development, Vol: 25, Pages: 382-391, ISSN: 2329-0501
We have developed a novel lentiviral vector, pseudotyped with the F and HN proteins from Sendai virus (rSIV.F/HN), which produces long-lasting, high efficiency transduction of the respiratory epithelium. Here, we addressed whether this platform technology can secrete sufficient levels of a therapeutic protein into the lung to ameliorate a fatal pulmonary disease, as an exemplar of its translational capability. Pulmonary Alveolar Proteinosis (PAP) results from alveolar GM-CSF insufficiency, resulting in abnormal surfactant homeostasis and consequent ventilatory problems. Lungs of GM-CSF knockout mice were transduced with a single dose of rSIV.F/HN expressing murine (m)GM-CSF (1e5-92e7 TU/mouse); mGM-CSF expression was dose-related and persisted for at least 11 months. PAP disease biomarkers were rapidly and persistently corrected, but we noted a narrow toxicity/efficacy window. rSIV.F/HN may be a useful platform technology to deliver therapeutic proteins for lung diseases requiring long-lasting and stable expression of secreted proteins.
Clarke NK, Sergijenko A, Pineault K, et al., 2022, Ex Vivo Transduced Macrophages Engraft in the Lung Following Transplantation and Produce Therapeutic Levels of Secreted Proteins, American Society of Cell and Gene Therapy, Publisher: CELL PRESS, Pages: 405-405, ISSN: 1525-0016
Bell R, Clarke NK, Isalan M, et al., 2022, Regulated Expression of LentiviralVectors Following Administration of anInducing Molecule, 2022 ASGCT Annual Meeting, Publisher: Cell Press, Pages: 419-419, ISSN: 1525-0016
Hickmott JW, Prasertsuk P, Bell RV, et al., 2022, Heterologous Signal Peptides Enhance Protein Secretion for Respiratory Gene Therapy, Publisher: CELL PRESS, Pages: 417-418, ISSN: 1525-0016
Sergijenko A, Pineault KM, Moiseenko A, et al., 2022, Development of Molecular Assays to Determine Lentiviral Vector-Mediated Transduction Efficiency in Airway Epithelial Cells, Publisher: CELL PRESS, Pages: 419-419, ISSN: 1525-0016
Bell RV, Clarke NK, Isalan M, et al., 2022, Regulated Expression of Lentiviral Vectors Following Administration of an Inducing Molecule, Publisher: CELL PRESS, Pages: 419-419, ISSN: 1525-0016
Clarke N, Sinadinos A, Meng C, et al., 2021, Ex vivo transduced macrophages produce therapeutic levels of secreted protein when transplanted to the lung, ESGCT Collaborative Virtual Congress, Publisher: Mary Ann Liebert, ISSN: 1043-0342
Bell R, Faulkner N, Griesenbach U, et al., 2021, Intravenous administration of F/HN pseudotyped lentiviral vector, ESGCT Collaborative Virtual Congress, Publisher: Mary Ann Liebert, ISSN: 1043-0342
Bell RV, Isalan M, Alton EWFW, et al., 2021, Regulation of lentivirus‐mediated expression in a human airway model, ESGCT Collaborative Virtual Congress, Publisher: Mary Ann Liebert, ISSN: 1043-0342
Griesenbach U, Yanez-Munoz RJ, 2021, The British Society for Gene and Cell Therapy, HUMAN GENE THERAPY, ISSN: 1043-0342
, 2021, ESGCTCollaborative Virtual Congress19–22 October 2021Abstracts, Human Gene Therapy, Vol: 32, Pages: A1-A152, 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, Publisher: MARY ANN LIEBERT, INC, Pages: A38-A38, ISSN: 1043-0342
Hickmott JW, Prasertsuk P, Bell RV, et al., 2021, Modifying signal peptides for respiratory gene therapy with secreted proteins, 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, 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, Publisher: MARY ANN LIEBERT, INC, Pages: A108-A109, ISSN: 1043-0342
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
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
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