The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice
Hordeaux, Juliette, Wang, Qiang, Katz, Nathan, Buza, Elizabeth L., Bell, Peter, Wilson, James M.
Published in Molecular therapy (07.03.2018)
Published in Molecular therapy (07.03.2018)
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Journal Article
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
Hordeaux, Juliette, Buza, Elizabeth L, Jeffrey, Brianne, Song, Chunjuan, Jahan, Tahsin, Yuan, Yuan, Zhu, Yanqing, Bell, Peter, Li, Mingyao, Chichester, Jessica A, Calcedo, Roberto, Wilson, James M
Published in Science translational medicine (11.11.2020)
Published in Science translational medicine (11.11.2020)
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Journal Article
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
Hordeaux, Juliette, Buza, Elizabeth L, Dyer, Cecilia, Goode, Tamara, Mitchell, Thomas W, Richman, Laura, Denton, Nathan, Hinderer, Christian, Katz, Nathan, Schmid, Ralf, Miller, Rod, Choudhury, Gourav R, Horiuchi, Makoto, Nambiar, Kalyani, Yan, Hanying, Li, Mingyao, Wilson, James M
Published in Human gene therapy (01.08.2020)
Published in Human gene therapy (01.08.2020)
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Journal Article
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys
Hordeaux, Juliette, Hinderer, Christian, Buza, Elizabeth L, Louboutin, Jean-Pierre, Jahan, Tahsin, Bell, Peter, Chichester, Jessica A, Tarantal, Alice F, Wilson, James M
Published in Human gene therapy (01.08.2019)
Published in Human gene therapy (01.08.2019)
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Journal Article
Efficacy and Safety of a Krabbe Disease Gene Therapy
Hordeaux, Juliette, Jeffrey, Brianne A, Jian, Jinlong, Choudhury, Gourav R, Michalson, Kristofer, Mitchell, Thomas W, Buza, Elizabeth L, Chichester, Jessica, Dyer, Cecilia, Bagel, Jessica, Vite, Charles H, Bradbury, Allison M, Wilson, James M
Published in Human gene therapy (01.05.2022)
Published in Human gene therapy (01.05.2022)
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Journal Article
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
Hordeaux, Juliette, Hinderer, Christian, Goode, Tamara, Katz, Nathan, Buza, Elizabeth L., Bell, Peter, Calcedo, Roberto, Richman, Laura K., Wilson, James M.
Published in Molecular therapy. Methods & clinical development (21.09.2018)
Published in Molecular therapy. Methods & clinical development (21.09.2018)
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Journal Article
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase
Hordeaux, Juliette, Ramezani, Ali, Tuske, Steve, Mehta, Nickita, Song, Chunjuan, Lynch, Anna, Lupino, Katherine, Chichester, Jessica A, Buza, Elizabeth L, Dyer, Cecilia, Yu, Hongwei, Bell, Peter, Weimer, Jill M, Do, Hung, Wilson, James M
Published in Frontiers in immunology (22.03.2023)
Published in Frontiers in immunology (22.03.2023)
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Journal Article
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques
Hordeaux, Juliette, Hinderer, Christian, Goode, Tamara, Buza, Elizabeth L., Bell, Peter, Calcedo, Roberto, Richman, Laura K., Wilson, James M.
Published in Molecular therapy. Methods & clinical development (21.09.2018)
Published in Molecular therapy. Methods & clinical development (21.09.2018)
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Journal Article
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Bey, Karim, Deniaud, Johan, Dubreil, Laurence, Joussemet, Béatrice, Cristini, Joseph, Ciron, Carine, Hordeaux, Juliette, Le Boulc’h, Morwenn, Marche, Kevin, Maquigneau, Maud, Guilbaud, Michaël, Moreau, Rosalie, Larcher, Thibaut, Deschamps, Jack-Yves, Fusellier, Marion, Blouin, Véronique, Sevin, Caroline, Cartier, Nathalie, Adjali, Oumeya, Aubourg, Patrick, Moullier, Philippe, Colle, Marie-Anne
Published in Molecular therapy. Methods & clinical development (12.06.2020)
Published in Molecular therapy. Methods & clinical development (12.06.2020)
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Journal Article
Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy
Rojsajjakul, Teerapat, Hordeaux, Juliette J., Choudhury, Gourav R., Hinderer, Christian J., Mesaros, Clementina, Wilson, James M., Blair, Ian A.
Published in Communications biology (27.10.2023)
Published in Communications biology (27.10.2023)
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Journal Article
Dose escalation study to evaluate safety, tolerability and efficacy of intravenous etoposide phosphate administration in 27 dogs with multicentric lymphoma
Boyé, Pierre, Serres, François, Marescaux, Laurent, Hordeaux, Juliette, Bouchaert, Emmanuel, Gomes, Bruno, Tierny, Dominique
Published in PloS one (15.05.2017)
Published in PloS one (15.05.2017)
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Journal Article
Assessment of adeno-associated virus gene therapies efficacy on acid alpha-glucosidase restoration and glycogen storage correction in cardiac muscle of Pompe disease mice using synchrotron infrared and ultraviolet microspectroscopies
Dubreil, Laurence, Lagalice, Lydie, Deniaud, Johan, Sabourin, Antoine, Lovo, Claire, Bey, Karim, Hordeaux, Juliette, Thorin, Chantal, Sandt, Christophe, Jamme, Frédéric, Colle, Marie-Anne
Published in Journal of spectral imaging (05.08.2019)
Published in Journal of spectral imaging (05.08.2019)
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Journal Article
The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier
Hordeaux, Juliette, Yuan, Yuan, Clark, Peter M., Wang, Qiang, Martino, R. Alexander, Sims, Joshua J., Bell, Peter, Raymond, Angela, Stanford, William L., Wilson, James M.
Published in Molecular therapy (08.05.2019)
Published in Molecular therapy (08.05.2019)
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Journal Article