Zobrazeno 1 - 10
of 21
pro vyhledávání: '"Anne Douar"'
Autor:
Oihana Murillo, Maria Collantes, Cristina Gazquez, Daniel Moreno, Ruben Hernandez-Alcoceba, Miren Barberia, Margarita Ecay, Blanche Tamarit, Anne Douar, Veronica Ferrer, Jean Philippe Combal, Ivan Peñuelas, Bernard Bénichou, Gloria Gonzalez-Aseguinolaza
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 98-106 (2022)
Wilson’s disease (WD) is an inherited disorder of copper metabolism associated with mutations in ATP7B gene. We have shown that the administration of an adeno-associated vector (AAV) encoding a mini version of human ATP7B (VTX-801) provides long-te
Externí odkaz:
https://doaj.org/article/afe87d2e90484e9ca008bbd452940d81
Autor:
Gregory Gauvain, Himanshu Akolkar, Antoine Chaffiol, Fabrice Arcizet, Mina A. Khoei, Mélissa Desrosiers, Céline Jaillard, Romain Caplette, Olivier Marre, Stéphane Bertin, Claire-Maelle Fovet, Joanna Demilly, Valérie Forster, Elena Brazhnikova, Philippe Hantraye, Pierre Pouget, Anne Douar, Didier Pruneau, Joël Chavas, José-Alain Sahel, Deniz Dalkara, Jens Duebel, Ryad Benosman, Serge Picaud
Publikováno v:
Communications Biology, Vol 4, Iss 1, Pp 1-15 (2021)
Gauvain et al demonstrate that optogenetic therapy using the AAV2.7m8- ChR-tdT construct can partially restore vision in non-human primates to levels above those considered legally-blind. This study enables the identification of the most suitable con
Externí odkaz:
https://doaj.org/article/0b4ca90d06f64da69afaff7c49d68f90
Autor:
Irene Ros‐Gañán, Mirja Hommel, Laia Trigueros‐Motos, Blanche Tamarit, Estefanía Rodríguez‐García, David Salas, Guiomar Pérez, Anne Douar, Jean Philippe Combal, Bernard Benichou, Veronica Ferrer, Gloria González‐Aseguinolaza
Publikováno v:
Clinical & Translational Immunology, Vol 11, Iss 2, Pp n/a-n/a (2022)
Abstract Objective Pre‐existing neutralising antibodies (NAbs) to adeno‐associated viruses (AAVs) remain an impediment for systemically administered AAV‐mediated gene therapy treatment in many patients, and various strategies are under investig
Externí odkaz:
https://doaj.org/article/df0f028936c04e56bad7ce9eb7f021f1
Autor:
Nicholas D. Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-13 (2019)
Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevent
Externí odkaz:
https://doaj.org/article/01e5e4ee2f0f4ae9875e80c35af9e27b
Autor:
Michael Martinez, Nicholas D. Weber, William Cao, Lori G Hillin, Robert A. Kaiser, Anne Douar, Kari L. Allen, Joseph B. Lillegard, Caitlin J. VanLith, Rafael Aldabe, Laia Trigueros-Motos, Gloria González-Aseguinolaza
Publikováno v:
Journal of Inherited Metabolic Disease. 44:1369-1381
Phenylketonuria (PKU) is the most common inborn error of metabolism of the liver, and results from mutations of both alleles of the phenylalanine hydroxylase gene (PAH). As such, it is a suitable target for gene therapy via gene delivery with a recom
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f23c76d8f40370a31f5d41f8847fe54e
https://doi.org/10.1002/jimd.12392
https://doi.org/10.1002/jimd.12392
Autor:
Oihana, Murillo, Maria, Collantes, Cristina, Gazquez, Daniel, Moreno, Ruben, Hernandez-Alcoceba, Miren, Barberia, Margarita, Ecay, Blanche, Tamarit, Anne, Douar, Veronica, Ferrer, Jean Philippe, Combal, Ivan, Peñuelas, Bernard, Bénichou, Gloria, Gonzalez-Aseguinolaza
Publikováno v:
Molecular therapy. Methodsclinical development. 26
Wilson's disease (WD) is an inherited disorder of copper metabolism associated with mutations in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::5fc5891b0cadc639f9fbb629b6de290c
https://pubmed.ncbi.nlm.nih.gov/35795774
https://pubmed.ncbi.nlm.nih.gov/35795774
Autor:
José-Alain Sahel, Fabrice Arcizet, Philippe Hantraye, Serge Picaud, Gregory Gauvain, Claire-Maëlle Fovet, Jens Duebel, Pierre Pouget, Mina A. Khoei, Deniz Dalkara, Joël Chavas, Melissa Desrosiers, Elena Brazhnikova, Ryad Benosman, Didier Pruneau, Olivier Marre, Antoine Chaffiol, Joanna Demilly, Himanshu Akolkar, Anne Douar, Valérie Forster, Romain Caplette, Céline Jaillard, Stéphane Bertin
Restoring vision using optogenetics is an ideal medical application because the eye offers a direct window to access and stimulate the pathological area: the retina. Optogenetic therapy could be applied to diseases with photoreceptor degeneration suc
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::e8d29dcc167fa17ca377fe8e3ece29fe
https://doi.org/10.1101/2020.05.17.100230
https://doi.org/10.1101/2020.05.17.100230
Autor:
Céline Jaillard, Gregory Gauvain, Serge Picaud, Olivier Marre, Antoine Chaffiol, Fabrice Arcizet, Stéphane Bertin, Pierre Pouget, Jens Duebel, Didier Pruneau, Anne Douar, Joël Chavas, Romain Caplette, Joanna Demilly, Elena Brazhnikova, Philippe Hantraye, Melissa Desrosiers, Valérie Forster, Claire-Maëlle Fovet, Mina A. Khoei, José-Alain Sahel, Himanshu Akolkar, Deniz Dalkara, Ryad Benosman
Publikováno v:
Communications Biology
Communications Biology, Nature Publishing Group, 2021, 4 (1), pp.125. ⟨10.1038/s42003-020-01594-w⟩
Communications Biology, 2021, 4 (1), pp.125. ⟨10.1038/s42003-020-01594-w⟩
Communications Biology, Vol 4, Iss 1, Pp 1-15 (2021)
Communications Biology, Nature Publishing Group, 2021, 4 (1), pp.125. ⟨10.1038/s42003-020-01594-w⟩
Communications Biology, 2021, 4 (1), pp.125. ⟨10.1038/s42003-020-01594-w⟩
Communications Biology, Vol 4, Iss 1, Pp 1-15 (2021)
Vision restoration is an ideal medical application for optogenetics, because the eye provides direct optical access to the retina for stimulation. Optogenetic therapy could be used for diseases involving photoreceptor degeneration, such as retinitis
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a5ff23b197250f56ca9445dc53cb323f
https://pubmed.ncbi.nlm.nih.gov/33504896
https://pubmed.ncbi.nlm.nih.gov/33504896
Autor:
Anne Douar, Javier Martínez-García, Bernard Bénichou, Nicholas D. Weber, Gloria González-Aseguinolaza, Cristian Smerdou, Veronica Ferrer, Leticia Odriozola
Publikováno v:
Nature Communications
Nature Communications, Vol 10, Iss 1, Pp 1-13 (2019)
Dadun. Depósito Académico Digital de la Universidad de Navarra
instname
Nature Communications, Vol 10, Iss 1, Pp 1-13 (2019)
Dadun. Depósito Académico Digital de la Universidad de Navarra
instname
Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare monogenic disease caused by mutations in the ABCB4 gene, resulting in a reduction in biliary phosphatidylcholine. Reduced biliary phosphatidylcholine cannot counteract the deterge
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b932561ebcad26b2df86cb61c44d43d7
https://doi.org/10.1038/s41467-019-13614-3
https://doi.org/10.1038/s41467-019-13614-3
Autor:
Veronica Ferrer, Gloria González-Aseguinolaza, Anne Douar, Javier Martínez-García, Cristian Smerdou, Leticia Odriozola, Nicholas DWeber, Bernard Bénichou
Publikováno v:
Journal of Hepatology. 70:e590
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::d90c9c395f9bbe3153e91db8b2f2e0cf
https://doi.org/10.1016/s0618-8278(19)31181-8
https://doi.org/10.1016/s0618-8278(19)31181-8