Zobrazeno 1 - 10
of 41
pro vyhledávání: '"Xavier M. Anguela"'
Autor:
Liron Elkouby, Sean M. Armour, Raffaella Toso, Marti DiPietro, Robert J. Davidson, Giang N. Nguyen, Mallory Willet, Stephanie Kutza, Joseph Silverberg, Jennifer Frick, Marco Crosariol, Yuhuan Wang, Chuansong Wang, Katherine A. High, Denise E. Sabatino, Xavier M. Anguela
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 20-29 (2022)
Extensive clinical data from liver-mediated gene therapy trials have shown that dose-dependent immune responses against the vector capsid may impair or even preclude transgene expression if not managed successfully with prompt immune suppression. The
Externí odkaz:
https://doaj.org/article/8d15f9b5838e44d49c75621a886fa6f5
Autor:
Helena Costa-Verdera, Fanny Collaud, Christopher R. Riling, Pauline Sellier, Jayme M. L. Nordin, G. Michael Preston, Umut Cagin, Julien Fabregue, Simon Barral, Maryse Moya-Nilges, Jacomina Krijnse-Locker, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jeremie Cosette, Catalina Abad, Marcelo Simon-Sola, Severine Charles, Mathew Li, Marco Crosariol, Tom Antrilli, William J. Quinn, David A. Gross, Olivier Boyer, Xavier M. Anguela, Sean M. Armour, Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, result
Externí odkaz:
https://doaj.org/article/ecdd7a66d5fe4b0e825ff6e616f835cf
Autor:
Chenhui Zou, Koen O.A. Vercauteren, Eleftherios Michailidis, Mohammad Kabbani, Irene Zoluthkin, Corrine Quirk, Luis Chiriboga, Mustafa Yazicioglu, Xavier M. Anguela, Philip Meuleman, Katherine A. High, Roland W. Herzog, Ype P. de Jong
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 189-198 (2020)
Adeno-associated virus (AAV) vector serotypes vary in their ability to transduce hepatocytes from different species. Chimeric mouse models harboring human hepatocytes have shown translational promise for liver-directed gene therapies. However, many v
Externí odkaz:
https://doaj.org/article/76e8a94fb75547cf982881d7ea650537
Autor:
Mohammad Kabbani, Philip Meuleman, Eleftherios Michailidis, Ype P. de Jong, Corrine Quirk, Chenhui Zou, Luis Chiriboga, Katherine A. High, Mustafa N. Yazicioglu, Koen Vercauteren, Roland W. Herzog, Xavier M. Anguela, Irene Zoluthkin
Publikováno v:
Molecular Therapy. Methods & Clinical Development
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 189-198 (2020)
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 189-198 (2020)
Adeno-associated virus (AAV) vector serotypes vary in their ability to transduce hepatocytes from different species. Chimeric mouse models harboring human hepatocytes have shown translational promise for liver-directed gene therapies. However, many v
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::425ecf07b20f36a97b45039f547d4ad7
http://europepmc.org/articles/PMC7326722
http://europepmc.org/articles/PMC7326722
Autor:
Xavier M Anguela, Kristen Jaworski, Stacy E Croteau, John E J Rasko, Tiffany Chang, Federico Mingozzi, Paul E Monahan, Katherine A. High, Kathleen Z Reape, Margaret V Ragni, Lindsey A. George, Amy Macdougall, Spencer K. Sullivan, M Elaine Eyster, Benjamin J. Samelson-Jones, Robert Noble, Michael Recht, Marla Curran, Klaudia Kuranda
Publikováno v:
N Engl J Med
Background The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. Methods In this phase 1-2 trial, we
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a13db718b1db9c4d1a4f5c07a1a3abc2
https://pubmed.ncbi.nlm.nih.gov/34788507
https://pubmed.ncbi.nlm.nih.gov/34788507
Autor:
Catalina Abad, N. Danièle, Jayme M.L. Nordin, Giuseppe Ronzitti, Bernard Gjata, Helena Costa-Verdera, Simon Barral, Sean M. Armour, Marcelo Simon-Sola, Fanny Collaud, Marco Crosariol, Julien Fabregue, David A. Gross, Mathew Li, Jérémie Cosette, Laetitia van Wittenberghe, Pasqualina Colella, Maryse Moya-Nilges, G. Michael Preston, Christopher Riling, Xavier M. Anguela, Federico Mingozzi, Severine Charles, Pauline Sellier, Tom Antrilli, William J. Quinn, Umut Cagin, Jacomina Krijnse-Locker, Olivier Boyer
Publikováno v:
Nature Communications
Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
Nature Communications, Nature Publishing Group, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
Nature Communications, Nature Publishing Group, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Pompe disease (PD) is a severe neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). PD is currently treated with enzyme replacement therapy (ERT) with intravenous infusions of recombinant human GAA (rhGAA)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9a1664cfde76136fe6521768528975a1
https://hal.sorbonne-universite.fr/hal-03449262/document
https://hal.sorbonne-universite.fr/hal-03449262/document
Autor:
Xavier M. Anguela, Katherine A. High
Publikováno v:
Annual Review of Medicine. 70:273-288
Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. Investigators are advancing with cautious optimism that effective, durable, and safe therapies
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5720c1d65c6e7827cdf0b1e2508d406d
https://doi.org/10.1146/annurev-med-012017-043332
https://doi.org/10.1146/annurev-med-012017-043332
Autor:
Margareth C. Ozelo, Katie Wachtel, Yifeng Chen, Benjamin J. Samelson-Jones, Leslie Raffini, Katherine A. High, Margaret V. Ragni, John E.J. Rasko, Xavier M. Anguela, Lindsey A. George, Jennifer Wellman, Alexa R. Runowski, Federico Mingozzi, Maria Hazbon, Klaudia Kuranda
Publikováno v:
Mol Ther
Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durabilit
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4e4ea62b5161a331b808e5546cb819c4
https://pubmed.ncbi.nlm.nih.gov/32559433
https://pubmed.ncbi.nlm.nih.gov/32559433
Autor:
Xavier M. Anguela, Katherine A. High
Publikováno v:
Research and Practice in Thrombosis and Haemostasis
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::165a28d3cd8688f01773bd88d29ec6e1
https://doi.org/10.1002/rth2.12190
https://doi.org/10.1002/rth2.12190
Autor:
Adeline Miranda, Dan Lupo, Federico Mingozzi, Xavier M. Anguela, Joseph Silverberg, Karen Huang, Heena Beck, Saghana Muraleetharan, Béatrice Marolleau, Fanny Collaud, Laetitia van Wittengerghe, Sean M. Armour, Christian Leborgne, Elena Barbon, Sandrine Delignat, Jeffrey M. Alexander, Hayley Hanby, Victoria Daventure, Giuseppe Ronzitti, Sébastien Lacroix-Desmazes, Anna Fabiano, Daniel M. Cohen
Publikováno v:
Nature Medicine
Nature Medicine, Nature Publishing Group, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Nature Medicine, Nature Publishing Group, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Neutralizing antibodies to adeno-associated virus (AAV) vectors are highly prevalent in humans1,2, and block liver transduction3–5 and vector readministration6; thus, they represent a major limitation to in vivo gene therapy. Strategies aimed at ov
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ff04724b6af379a7e9370adc25a9aa43
https://hal.archives-ouvertes.fr/hal-03031762
https://hal.archives-ouvertes.fr/hal-03031762