Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

Autor: Rosalie Moreau, Carine Ciron, Nathalie Cartier, Oumeya Adjali, Jack-Yves Deschamps, Béatrice Joussemet, Thibaut Larcher, Morwenn Le Boulc’h, Kevin Marche, Véronique Blouin, Joseph Cristini, Marie-Anne Colle, Patrick Aubourg, Karim Bey, Maud Maquigneau, Caroline Sevin, Juliette Hordeaux, Marion Fusellier, Laurence Dubreil, Michaël Guilbaud, Johan Deniaud, Philippe Moullier

Publikováno v: Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, 2020, 17, pp.771-784. ⟨10.1016/j.omtm.2020.04.001⟩
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2020, 17, pp.771-784. ⟨10.1016/j.omtm.2020.04.001⟩
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 771-784 (2020)
Molecular Therapy. Methods & Clinical Development

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://explore.openaire.eu/search/publication?articleId=doi_dedup___::f92dd8326fe4a471d375159eed1bd5bc
https://hal.inrae.fr/hal-02561949

Gene-Gun Biolistic Immunization Encoding Glutamic Acid Decarboxylase: A Model for Studying Langerhans Cell Abnormalities and Mimicry in the Nonobese Diabetic Mouse

Autor: Béatrice Joussemet, Anh-Tuan Vu, Jean-Marie Bach, Pierre Sai

Publikováno v: Annals of the New York Academy of Sciences
Annals of the New York Academy of Sciences, Wiley, 2005, 1051, pp.613-625. ⟨10.1196/annals.1361.106⟩

Plasmid-DNA gene-gun immunization may be an efficient approach for investigating the role of skin dendritic cells (DCs) in type 1 diabetes (T1D) pathogenesis and the significance of the presentation of peptides that mimic autoantigenic epitopes in ag

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6b647f4e883cd9499d65f66016caa965
https://doi.org/10.1196/annals.1361.106

Safe, efficient, and reproductible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes

Autor: Sylvie Raoul, Yan Cherel, Béatrice Joussemet, Stéphanie Bigou, Jean-Michel Heard, Karen L. Kline, Philippe Moullier, Jérôme Ausseil, Song Liu, Monique Piraud, Eman E.A. Mohammed, Irène Maire, Christopher B. Thomson, N. Matthew Ellinwood, Marie-Thérèse Vanier, Jackie K. Jens, Nathalie Desmaris, Harald Petry, Jennifer D. Parkes, Stephan Hermening, Elizabeth M. Snella, Rachid Benchaouir, Roseline Froissart, Yaouen Lajat, Marie-Anne Colle, Marc Tardieu, Carine Ciron, Françoise A. Roux

Publikováno v: Molecular Therapy
Molecular Therapy, 2011, 19 (2), pp.251-259. ⟨10.1038/mt.2010.265⟩
Molecular Therapy, Cell Press, 2011, 19 (2), pp.251-259. ⟨10.1038/mt.2010.265⟩

International audience; Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent caus

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ebd8d6e36d1785e0f293b78db4eaf5b0
https://hal.inrae.fr/hal-02652125

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

Autor: Martine Barkats, S. Duque, Philippe Moullier, Marie Anne Colle, Béatrice Joussemet, Laurence Dubreil, Anne Douar, John C. Fyfe, Christel Rivière, Thibaut Marais

Publikováno v: Molecular Therapy
Molecular Therapy, 2009, 17 (7), pp.1187-1196. ⟨10.1038/mt.2009.71⟩
Molecular Therapy, Cell Press, 2009, 17 (7), pp.1187-1196. ⟨10.1038/mt.2009.71⟩

International audience; Therapeutic gene delivery to the whole spinal cord is a major challenge for the treatment of motor neuron (MN) diseases. Systemic administration of viral gene vectors would provide an optimal means for the long-term delivery o

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0420350a438f0da8b3ca3317683d925c
https://hal.inrae.fr/hal-02666576

Optimal design of a single recombinant adeno-associated virus derived from serotypes 1 and 2 to achieve more tightly regulated transgene expression from nonhuman primate muscle

Autor: Thibaut Larcher, David Favre, Philippe Moullier, Pierre Chenuaud, Hermann Bujard, Joseph E. Rabinowitz, Béatrice Joussemet, Nathalie Provost, Richard Jude Samulski

Publikováno v: Molecular Therapy
Molecular Therapy, Cell Press, 2004, 9 (3), pp.410-418. ⟨10.1016/j.ymthe.2003.12.015⟩

International audience; Recombinant adeno-associated virus (rAAV) vector supports long-term transgene expression from skeletal muscle in most mammals, including human. In some instances, the requirement for tight control of the transgene expression i

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9c07f45519ebb775419872463775a4f3
https://hal.inrae.fr/hal-02678459