Zobrazeno 1 - 10
of 31
pro vyhledávání: '"Juliette Hordeaux"'
Autor:
Juliette Hordeaux, Ali Ramezani, Steve Tuske, Nickita Mehta, Chunjuan Song, Anna Lynch, Katherine Lupino, Jessica A. Chichester, Elizabeth L. Buza, Cecilia Dyer, Hongwei Yu, Peter Bell, Jill M. Weimer, Hung Do, James M. Wilson
Publikováno v:
Frontiers in Immunology, Vol 14 (2023)
Immune responses to human non-self transgenes can present challenges in preclinical studies of adeno-associated virus (AAV) gene therapy candidates in nonhuman primates. Although anti-transgene immune responses are usually mild and non-adverse, they
Externí odkaz:
https://doaj.org/article/399a575278114cbda824ff19d97176ed
Autor:
Karim Bey, Johan Deniaud, Laurence Dubreil, Béatrice Joussemet, Joseph Cristini, Carine Ciron, Juliette Hordeaux, Morwenn Le Boulc’h, Kevin Marche, Maud Maquigneau, Michaël Guilbaud, Rosalie Moreau, Thibaut Larcher, Jack-Yves Deschamps, Marion Fusellier, Véronique Blouin, Caroline Sevin, Nathalie Cartier, Oumeya Adjali, Patrick Aubourg, Philippe Moullier, Marie-Anne Colle
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 771-784 (2020)
The identification of the most efficient method for whole central nervous system targeting that is translatable to humans and the safest route of adeno-associated virus (AAV) administration is a major concern for future applications in clinics. Addit
Externí odkaz:
https://doaj.org/article/a0aedb1aca064422b991e72f09f1cc8d
Autor:
Juliette Hordeaux, Christian Hinderer, Tamara Goode, Nathan Katz, Elizabeth L. Buza, Peter Bell, Roberto Calcedo, Laura K. Richman, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 79-88 (2018)
Mucopolysaccharidosis type I is a recessive genetic disease caused by deficiency of the lysosomal enzyme α-L-iduronidase, which leads to a neurodegenerative and systemic disease called Hurler syndrome in its most severe form. Several clinical trials
Externí odkaz:
https://doaj.org/article/1f37efe1f3c9466eaf96059d5c8d3fc8
Autor:
Juliette Hordeaux, Christian Hinderer, Tamara Goode, Elizabeth L. Buza, Peter Bell, Roberto Calcedo, Laura K. Richman, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 68-78 (2018)
Hunter syndrome is an X-linked recessive disease caused by deficiency of the lysosomal enzyme iduronate-2-sulfatase. The severe form of this progressive, systemic, and neurodegenerative disease results in loss of cognitive skills and early death. Sev
Externí odkaz:
https://doaj.org/article/5a204333500b4ac4af8e689cf632c042
Autor:
Laurence Dubreil, Lydie Lagalice, Johan Deniaud, Antoine Sabourin, Claire Lovo, Karim Bey, Juliette Hordeaux, Chantal Thorin, Christophe Sandt, Frédéric Jamme, Marie-Anne Colle
Publikováno v:
Journal of Spectral Imaging, Vol 8, Iss 1, p a13 (2019)
Pompe disease (glycogen storage disease type II) is a lysosomal storage disorder due to a mutation in the gene that encodes acid alpha-glucosidase (GAA). GAA deficiency causes the excessive storage of lysosomal glycogen in many cell types, leading to
Externí odkaz:
https://doaj.org/article/c43b1b00ac0642fcaaad362ed97e6ee1
Autor:
Pierre Boyé, François Serres, Laurent Marescaux, Juliette Hordeaux, Emmanuel Bouchaert, Bruno Gomes, Dominique Tierny
Publikováno v:
PLoS ONE, Vol 12, Iss 5, p e0177486 (2017)
Comparative oncology has shown that naturally occurring canine cancers are of valuable and translatable interest for the understanding of human cancer biology and the characterization of new therapies. This work was part of a comparative oncology pro
Externí odkaz:
https://doaj.org/article/d11316accbc742efa438934ac6786121
Autor:
Juliette Hordeaux, Brianne A. Jeffrey, Jinlong Jian, Gourav R. Choudhury, Kristofer Michalson, Thomas W. Mitchell, Elizabeth L. Buza, Jessica Chichester, Cecilia Dyer, Jessica Bagel, Charles H. Vite, Allison M. Bradbury, James M. Wilson
Publikováno v:
Human gene therapy. 33(9-10)
Krabbe disease is a lysosomal storage disease caused by mutations in the gene that encodes galactosylceramidase, in which galactosylsphingosine (psychosine) accumulation drives demyelination in the central and peripheral nervous systems, ultimately p
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::450f4a43e12fd74bd092d6865f466f03
https://pubmed.ncbi.nlm.nih.gov/35333110
https://pubmed.ncbi.nlm.nih.gov/35333110
Autor:
R. Alexander Martino, Peter Bell, Peter Clark, James M. Wilson, Angela Raymond, William L. Stanford, Yuan Yuan, Joshua J. Sims, Juliette Hordeaux, Qiang Wang
Publikováno v:
Molecular Therapy. 27:912-921
Efficient delivery of gene therapy vectors across the blood-brain barrier (BBB) is the holy grail of neurological disease therapies. A variant of the neurotropic vector adeno-associated virus (AAV) serotype 9, called AAV-PHP.B, was shown to very effi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::16c504be94b917aafbc27fde77e2d811
https://doi.org/10.1016/j.ymthe.2019.02.013
https://doi.org/10.1016/j.ymthe.2019.02.013
Autor:
Roberto Calcedo, Jessica A. Chichester, Peter Bell, Mingyao Li, Tahsin Jahan, Juliette Hordeaux, Yuan Yuan, Yanqing Zhu, Brianne A. Jeffrey, Elizabeth L. Buza, James M. Wilson, Chunjuan Song
Publikováno v:
Science Translational Medicine. 12
Delivering adeno-associated virus (AAV) vectors into the central nervous system of nonhuman primates (NHPs) via the blood or cerebral spinal fluid is associated with dorsal root ganglion (DRG) toxicity. Conventional immune-suppression regimens do not
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::092e80ad8233d4451c35bba334d390a1
https://doi.org/10.1126/scitranslmed.aba9188
https://doi.org/10.1126/scitranslmed.aba9188
Autor:
Nathan Denton, Gourav Roy Choudhury, Nathan Katz, Mingyao Li, Hanying Yan, Ralf Schmid, Laura K. Richman, Makoto Horiuchi, Rod Miller, Kalyani Nambiar, Juliette Hordeaux, Cecilia Dyer, Christian Hinderer, Elizabeth L. Buza, James M. Wilson, Tamara Goode, Thomas W. Mitchell
Publikováno v:
Human gene therapy. 31(15-16)
The administration of adeno-associated virus (AAV) vectors to nonhuman primates (NHP) via the blood or cerebrospinal fluid (CSF) can lead to dorsal root ganglion (DRG) pathology. The pathology is minimal to moderate in most cases; clinically silent i
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4fb824755de81f2c8bc9f20c7fd78cba
https://pubmed.ncbi.nlm.nih.gov/34283921
https://pubmed.ncbi.nlm.nih.gov/34283921