Lentiviral vector packaging and producer cell lines yield titers equivalent to the industry-standard four-plasmid process

Authors: Tridgett, Matthew, Mulet, Marie, Johny, Sherin Parokkaran, Ababi, Maria, Raghunath, Meenakshi, Fustinoni, Chloé, Galabova, Boryana, Fernández-Díaz, Cristina, Mikalajūnaitė, Iveta, Tomás, Hélio A., Kucej, Marek, Dunajová, Lucia, Zgrundo, Zofia, Page, Emma, McCall, Lorna, Parker-Manuel, Richard, Payne, Tom, Peckett, Matthew, Kent, Jade, Holland, Louise, Asatryan, Robert, Montgomery, Louise, Chow, Tsz Lung, Beveridge, Ryan, Salkauskaite, Ieva, Alam, Mohine T., Hollard, Daniel, Dowding, Sarah, Gabriel, Heloísa Berti, Branciaroli, Corinne, Cawood, Ryan, Valenti, Weimin, Chang, David, Patrício, Maria I., Liu, Qian

Source: In Molecular Therapy - Methods & Clinical Development 12 September 2024 32(3)

Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy

Authors: Patrício, Maria I., Cox, Christopher I., Blue, Clare, Barnard, Alun R., Martinez-Fernandez de la Camara, Cristina, MacLaren, Robert E.

Source: In Molecular Therapy - Methods & Clinical Development 12 June 2020 17:99-106

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

Authors: Chandler, Laurel C., Barnard, Alun R., Caddy, Sarah L., Patrício, Maria I., McClements, Michelle E., Fu, Howell, Rada, Cristina, MacLaren, Robert E., Xue, Kanmin

Source: In Molecular Therapy - Methods & Clinical Development 13 September 2019 14:77-89

Association of Messenger RNA Level With Phenotype in Patients With ChoroideremiaPotential Implications for Gene Therapy Dose

Authors: Fry, Lewis E., Patrício, Maria I., Williams, Jonathan

Source: JAMA ophthalmology. 138(2):128-135

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN633430263&indx=1&recIds=ETOCRN633430263

Effect of AAV-Mediated Rhodopsin Gene Augmentation on Retinal Degeneration Caused by the Dominant P23H Rhodopsin Mutation in a Knock-In Murine Model

Authors: Orlans, Harry O., Barnard, Alun R., Patrício, Maria I., McClements, Michelle E., MacLaren, Robert E.

Source: Human gene therapy. 31(13/14):730-742

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100152111428.0x000001&indx=1&recIds=ETOCvdc_100152111428.0x000001

The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A

Authors: Patrício, Maria I., Barnard, Alun R., Cox, Christopher I., Blue, Clare, MacLaren, Robert E.

Source: In Molecular Therapy - Methods & Clinical Development 15 June 2018 9:288-295

Atypical choroideremia presenting with early‐onset macular atrophy

Authors: Kontos, Georgios, Kwan, Jennifer, Xue, Kanmin, Patrício, Maria I., Clouston, Penny, Packham, Emily, MacLaren, Robert E., Downes, Susan M.

Source: Acta ophthalmologica. 97(6):633-636

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100086362410.0x000001&indx=1&recIds=ETOCvdc_100086362410.0x000001

A Quantitative Chloride Channel Conductance Assay for Efficacy Testing of AAV.BEST1

Authors: Wood, Shaun R., McClements, Michelle E., Martinez-Fernandez de la Camara, Cristina, Patrício, Maria I., Uggenti, Carolina, Sekaran, Sumathi, Barnard, Alun R., Manson, Forbes D., MacLaren, Robert E.

Source: Human gene therapy.Part B.Methods. 30(2):44-52

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100083621713.0x000001&indx=1&recIds=ETOCvdc_100083621713.0x000001

Progress in the development of novel therapies for choroideremia

Authors: Cehajic Kapetanovic, Jasmina, Patrício, Maria I, MacLaren, Robert E

Source: Expert review of ophthalmology. 14(6):277-285

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100096894394.0x000001&indx=1&recIds=ETOCvdc_100096894394.0x000001

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

Authors: Patrício, Maria I., Barnard, Alun R., Orlans, Harry O., McClements, Michelle E., MacLaren, Robert E.

Source: In Molecular Therapy - Nucleic Acids 17 March 2017 6:198-208

A clinical-grade gene therapy vector for pharmacoresistant epilepsy successfully overexpresses NPY in a human neuronal cell line

Authors: Patrício, Maria I., Barnard, Alun R., Green, Alexander L., During, Matthew J., Sen, Arjune, MacLaren, Robert E.

Source: Seizure. 55:25-29

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100055234544.0x000001&indx=1&recIds=ETOCvdc_100055234544.0x000001

Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy

Authors: Orlans, Harry O., Edwards, Thomas L., De Silva, Samantha R., Patrício, Maria I., MacLaren, Robert E.

Source: Methods in molecular biology. 1715:289-304

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN614054655&indx=1&recIds=ETOCRN614054655

Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector

Authors: Patrício, Maria I., MacLaren, Robert E.

Source: Methods in molecular biology. 1715:89-100

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN614054515&indx=1&recIds=ETOCRN614054515

Choroideremia: molecular mechanisms and development of AAV gene therapy

Authors: Patrício, Maria I, Barnard, Alun R, Xue, Kanmin, MacLaren, Robert E

Source: Expert opinion on biological therapy. 18(7):807-820

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100106500165.0x000001&indx=1&recIds=ETOCvdc_100106500165.0x000001

Retinal Degeneration in Choroideremia follows an Exponential Decay Function

Authors: Aylward, James W., Xue, Kanmin, Patrício, Maria I., Jolly, Jasleen K., Wood, Jonathan C., Brett, Jonathan, Jasani, Kirti M., MacLaren, Robert E.

Source: Ophthalmology. 125(7):1122-1124

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100065986068.0x000001&indx=1&recIds=ETOCvdc_100065986068.0x000001

Stroma-derived IL-6, G-CSF and Activin-A mediated dedifferentiation of lung carcinoma cells into cancer stem cells

Authors: Rodrigues, Carlos F. D., Serrano, Eurico, Patrício, Maria I., Val, Mariana M., Albuquerque, Patrícia, Fonseca, João, Gomes, Célia M. F., Abrunhosa, Antero J., Paiva, Artur, Carvalho, Lina, Botelho, M. Filomena, Almeida, Luís, Carreira, Isabel M., Alpoim, Maria Carmen

Source: Scientific reports. 8(1)

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100131211870.0x000001&indx=1&recIds=ETOCvdc_100131211870.0x000001

Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues

Authors: Chandler, Laurel C., Barnard, Alun R., Caddy, Sarah L., Patrício, Maria I., McClements, Michelle E., Fu, Howell, Rada, Cristina, MacLaren, Robert E., Xue, Kanmin

Source: In Molecular Therapy - Methods & Clinical Development 9 March 2023 28:300-300

Self-attenuating adenovirus enables production of recombinant adeno-associated virus for high manufacturing yield without contamination.

Authors: Su, Weiheng, Patrício, Maria I., Duffy, Margaret R., Krakowiak, Jakub M., Seymour, Leonard W., Cawood, Ryan

Source: Nature Communications; 3/17/2022, Vol. 13 Issue 1, p1-14, 14p

Subject Terms: ADENO-associated virus, RECOMBINANT viruses, ADENOVIRUSES, GENE amplification, GENE therapy, BINDING sites, TETRACYCLINE

Contributors

Authors: Aleman, Tomas S., Amati-Bonneau, Patrizia, Arveiler, Benoît, Ashworth, Jane L., Audo, Isabelle, Bacci, Giacomo M., Balducci, Nicole, Balikova, Irina, Bauwens, Miriam, Barboni, Piero, Birtel, Johannes, Biswas, Susmito, Black, Graeme C.M., Blanchet, Catherine, Bocquet, Béatrice, Boon, Camiel J.F., Brézin, Antoine, Roziers, Cyril Burin des, Burkitt-Wright, Emma, Callea, Michele, Carbonelli, Michele, Carelli, Valerio, Cehajic-Kapetanovic, Jasmina, Chandler, Kate E., Chandra, Aman, Clayton-Smith, Jill, Colijn, Johanna M., Coppieters, Frauke, A. Cukras, Catherine, Daly, Avril, De Baere, Elfride, De Zaeytijd, Julie, Borman, Arundhati Dev, Dollfus, Hélène, Houge, Sofia Douzgou, Engle, Elizabeth C., Escher, Pascal, Evans, D. Gareth, Fahnehjelm, Kristina Teär, Fasser, Christina, Fiore, Mathieu, Fujinami, Kaoru, Fujinami-Yokokawa, Yu, Gallie, Brenda L., Georgiou, Michalis, Gliem, Martin, Grudzinska Pechhacker, Monika K., Hall, Georgina, Harmening, Wolf M., Henderson, Robert H., Héon, Elise, Hirji, Nashila, Holz, Frank G., A. Huryn, Laryssa, Jones, Elizabeth A., Kalatzis, Vasiliki, Khan, Arif O., Kim, Ungsoo S., Klaver, Caroline C.W., Kumaran, Neruban, La Morgia, Chiara, Lalloo, Fiona, Lasseaux, Eulalie, Lee, Helena, Lenaers, Guy, Lenassi, Eva, Leroy, Bart P., Liskova, Petra, Lloyd, I. Christopher, MacLaren, Robert E., Mahroo, Omar A., Mejia-Vergara, Alvaro J., Meunier, Isabelle, Michaelides, Michel, Moore, Anthony T., Moosajee, Mariya, Morice-Picard, Fanny, Munier, Francis L., Neveu, Magella M., O'Neil, Erin C., Nordenström, Anna, Parry, Neil R.A., Patrício, Maria I., Parulekar, Manoj V., Ram, Dipak, Ramsden, Simon C., Robitaille, Johane, Robson, Anthony G., Rothschild, Pierre-Raphaël, Sadun, Alfredo A., Schuerch, Kaspar, Seabra, Miguel C., Self, Jay E., Sergouniotis, Panagiotis I., Shaya, Fadi, Sieving, Paul A., Strubbe, Ine, Simonelli, Francesca, Small, Kent W., Snead, Martin P., Stepien, Karolina M., Talib, Mays, Taylor, Rachel L., Testa, Francesco, Thiadens, Alberta A.H.J., Traboulsi, Elias I., Tran, Viet H., Vaclavik, Veronika, Valleix, Sophie, Van Cauwenbergh, Caroline, Van Schil, Kristof, Whitman, Mary C., Willoughby, Colin E., Xue, Kanmin, Yang, Jingyan, Yu-Wai-Man, Patrick, Zeitz, Christina, Zinkernagel, Martin

Source: In Clinical Ophthalmic Genetics and Genomics 2022:xi-xiv

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