Zobrazeno 1 - 10
of 32
pro vyhledávání: '"Maria I. Patrício"'
Autor:
Laurel C. Chandler, Alun R. Barnard, Sarah L. Caddy, Maria I. Patrício, Michelle E. McClements, Howell Fu, Cristina Rada, Robert E. MacLaren, Kanmin Xue
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 300- (2023)
Externí odkaz:
https://doaj.org/article/431c72f30eab4cbdaf74c062496480db
Autor:
Weiheng Su, Maria I. Patrício, Margaret R. Duffy, Jakub M. Krakowiak, Leonard W. Seymour, Ryan Cawood
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
Scalability, yield and quality of recombinant AAVs are a significant issue during manufacture. Here the authors describe a self-inhibiting helper plasmid strategy that improves outcomes compared to helper-free approaches.
Externí odkaz:
https://doaj.org/article/b9f6c18d18514eb388b17a1dd87ec0ce
Autor:
Maria I. Patrício, Christopher I. Cox, Clare Blue, Alun R. Barnard, Cristina Martinez-Fernandez de la Camara, Robert E. MacLaren
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 99-106 (2020)
Recent advances in recombinant adeno-associated virus (rAAV) gene therapy for choroideremia show gene replacement to be a promising approach. It is, however, well known that contact of vector solution with plastic materials in the surgical device may
Externí odkaz:
https://doaj.org/article/49c20b8bb5de44f498778030fc2b34c4
Autor:
Carlos F. D. Rodrigues, Eurico Serrano, Maria I. Patrício, Mariana M. Val, Patrícia Albuquerque, João Fonseca, Célia M. F. Gomes, Antero J. Abrunhosa, Artur Paiva, Lina Carvalho, M. Filomena Botelho, Luís Almeida, Isabel M. Carreira, Maria Carmen Alpoim
Publikováno v:
Scientific Reports, Vol 8, Iss 1, Pp 1-12 (2018)
Abstract Cancer stem cells (CSCs) are a small population of resistant cells inhabiting the tumors. Although comprising only nearly 3% of the tumor mass, these cells were demonstrated to orchestrate tumorigenesis and differentiation, underlie tumors
Externí odkaz:
https://doaj.org/article/6bb37cff1bbf4fc29ff5c570d306e16b
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 9, Iss , Pp 288-295 (2018)
Choroideremia (CHM) is a rare, X-linked recessive retinal dystrophy caused by mutations in the CHM gene. CHM is ubiquitously expressed in human cells and encodes Rab escort protein 1 (REP1). REP1 plays a key role in intracellular trafficking through
Externí odkaz:
https://doaj.org/article/c919bce748b94ee782f3543cc6ff781c
Autor:
Maria I. Patrício, Alun R. Barnard, Harry O. Orlans, Michelle E. McClements, Robert E. MacLaren
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 6, Iss , Pp 198-208 (2017)
The woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) has been included in the transgene cassette of adeno-associated virus (AAV) in several gene therapy clinical trials, including those for inherited retinal diseases. However,
Externí odkaz:
https://doaj.org/article/38656f8a3f8643848fee04932cec06f2
Publikováno v:
Human Gene Therapy. 31:730-742
Mutations in the rhodopsin gene may cause photoreceptor degeneration in autosomal dominant retinitis pigmentosa (ADRP) by dominant negative or toxic gain-of-function mechanisms. Controversy exists as to the mechanism by which the widely studied P23H
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b454dc8362bc367b2242460d6225b1d0
https://doi.org/10.1089/hum.2020.008
https://doi.org/10.1089/hum.2020.008
Autor:
Tomas S. Aleman, Patrizia Amati-Bonneau, Benoît Arveiler, Jane L. Ashworth, Isabelle Audo, Giacomo M. Bacci, Nicole Balducci, Irina Balikova, Miriam Bauwens, Piero Barboni, Johannes Birtel, Susmito Biswas, Graeme C.M. Black, Catherine Blanchet, Béatrice Bocquet, Camiel J.F. Boon, Antoine Brézin, Cyril Burin des Roziers, Emma Burkitt-Wright, Michele Callea, Michele Carbonelli, Valerio Carelli, Jasmina Cehajic-Kapetanovic, Kate E. Chandler, Aman Chandra, Jill Clayton-Smith, Johanna M. Colijn, Frauke Coppieters, Catherine A. Cukras, Avril Daly, Elfride De Baere, Julie De Zaeytijd, Arundhati Dev Borman, Hélène Dollfus, Sofia Douzgou Houge, Elizabeth C. Engle, Pascal Escher, D. Gareth Evans, Kristina Teär Fahnehjelm, Christina Fasser, Mathieu Fiore, Kaoru Fujinami, Yu Fujinami-Yokokawa, Brenda L. Gallie, Michalis Georgiou, Martin Gliem, Monika K. Grudzinska Pechhacker, Georgina Hall, Wolf M. Harmening, Robert H. Henderson, Elise Héon, Nashila Hirji, Frank G. Holz, Laryssa A. Huryn, Elizabeth A. Jones, Vasiliki Kalatzis, Arif O. Khan, Ungsoo S. Kim, Caroline C.W. Klaver, Neruban Kumaran, Chiara La Morgia, Fiona Lalloo, Eulalie Lasseaux, Helena Lee, Guy Lenaers, Eva Lenassi, Bart P. Leroy, Petra Liskova, I. Christopher Lloyd, Robert E. MacLaren, Omar A. Mahroo, Alvaro J. Mejia-Vergara, Isabelle Meunier, Michel Michaelides, Anthony T. Moore, Mariya Moosajee, Fanny Morice-Picard, Francis L. Munier, Magella M. Neveu, Erin C. O'Neil, Anna Nordenström, Neil R.A. Parry, Maria I. Patrício, Manoj V. Parulekar, Dipak Ram, Simon C. Ramsden, Johane Robitaille, Anthony G. Robson, Pierre-Raphaël Rothschild, Alfredo A. Sadun, Kaspar Schuerch, Miguel C. Seabra, Jay E. Self, Panagiotis I. Sergouniotis, Fadi Shaya, Paul A. Sieving, Ine Strubbe, Francesca Simonelli, Kent W. Small, Martin P. Snead, Karolina M. Stepien, Mays Talib, Rachel L. Taylor, Francesco Testa, Alberta A.H.J. Thiadens, Elias I. Traboulsi, Viet H. Tran, Veronika Vaclavik, Sophie Valleix, Caroline Van Cauwenbergh, Kristof Van Schil, Mary C. Whitman, Colin E. Willoughby, Kanmin Xue, Jingyan Yang, Patrick Yu-Wai-Man, Christina Zeitz, Martin Zinkernagel
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::443fea97f8aa209eb7749fe6b0bb2a99
https://doi.org/10.1016/b978-0-12-813944-8.09991-1
https://doi.org/10.1016/b978-0-12-813944-8.09991-1
Autor:
Weiheng, Su, Maria I, Patrício, Margaret R, Duffy, Jakub M, Krakowiak, Leonard W, Seymour, Ryan, Cawood
Publikováno v:
Nature communications. 13(1)
Recombinant adeno-associated virus (rAAV) shows great promise for gene therapy, however scalability, yield and quality remain significant issues. Here we describe an rAAV manufacturing strategy using a 'helper' adenovirus that self-inhibits its major
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::fa1b50925e690d21ef9eb35fea9f8dec
https://pubmed.ncbi.nlm.nih.gov/35256603
https://pubmed.ncbi.nlm.nih.gov/35256603
Autor:
Susan M. Downes, Penny Clouston, Jennifer Kwan, Kanmin Xue, Georgios T. Kontos, Maria I. Patrício, Robert E MacLaren, Emily Packham
Publikováno v:
Acta Ophthalmologica. 97:633-636
Choroideremia is an X-linked recessive retinal degeneration predominantly affecting hemizygous males. It is caused by mutations in the CHM gene that encodes the Rab escort protein-1. Characteristic features include early nyctalopia followed by progre
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e378181a05acb398754effbf1eea104d
https://doi.org/10.1111/aos.14025
https://doi.org/10.1111/aos.14025