Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome

Autor: Marie-Lise Gougeon, Béatrice Poirier-Beaudouin, Jérome Ausseil, Michel Zérah, Cécile Artaud, Jean-Michel Heard, Kumaran Deiva, Marc Tardieu

Publikováno v: Frontiers in Immunology, Vol 12 (2021)

Mucopolysaccharidosis type IIIB syndrome (Sanfilippo disease) is a rare autosomic recessif disorder caused by mutations in the α-N-acetylglucosaminidase (NAGLU) gene coding for a lysosomal enzyme, leading to neurodegeneration and progressive deterio

Externí odkaz: https://doaj.org/article/a77948f9fa2f41ada8f3d223aac4708b

Safety and efficacy of teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial

Autor: Tanuja Chitnis, Brenda Banwell, Ludwig Kappos, Douglas L Arnold, Kivilcim Gücüyener, Kumaran Deiva, Natalia Skripchenko, Li-Ying Cui, Stephane Saubadu, Wenruo Hu, Myriam Benamor, Annaig Le-Halpere, Philippe Truffinet, Marc Tardieu, Benedicte Dubois, Helene Verhelst, Veneta Bojinova-Tchamova, Jean Mah, Fang Fang, Yunpeng Hao, Li Jiang, Ling Li, Ding'An Mao, Wei Qiu, Guojun Tan, Ye Wu, Meini Zhang, Hongyu Zhou, Shuizhen Zhou, Katrin Gross-Paju, Emmanuel Cheuret, Giles Edan, Sandra Vukusic, George Chrousos, Dimitrios Zafeiriou, Anat Achiron, Adi Vaknin-Dembinsky, Bassem Yamout, Jurate Laurynaitiene, Nerija Vaiciene-Magistris, Vladimir Bojkovski, Vesna Trajkova, Sana Chaouki, Najib Kissani, Rinze Neuteboom, Filipe Palavra, Anna Belova, Alexey Boyko, Evgeny Evdoshenko, Ekaterina Kairbekova, Nadezhda Malkova, Maria Shumilina, Natalya Skripchenko, Dimitrije Nikolic, Jose Meca-Lallana, Chahnez Charfi Triki, Mhiri Chokri, Riadh Gouider, Banu Anlar, Ayse Semra Hiz, Egemen Idiman, Recai Turkoglu, Zuhal Yapici, Unsal Yilmaz, Lyudmyla Tantsura, Nataliia Voloshyna, Ming Lim, Evangeline Wassmer, Mark Cascione, Christopher LaGanke, Kevin Rathke, John Scagnelli

Publikováno v: The Lancet Neurology, 20(12), 1001-1011. Lancet Publishing Group

Background: Therapeutic options for children with multiple sclerosis are scarce. Teriflunomide is approved in more than 80 countries for the treatment of adults with relapsing multiple sclerosis. The TERIKIDS study examined the safety and efficacy of

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ccabed9102b9a00a1d52c5bf3629e63
https://pure.eur.nl/en/publications/9a1acc50-a431-44d3-9955-e7dcdc6b22c3

Taux plasmatique des neurofilaments à chaîne légère chez les enfants atteints de sclérose en plaques récurrente-rémittente traités par tériflunomide ou placebo : une analyse post hoc de l’essai randomisé TERIKIDS

Autor: Philippe Truffinet, Jens Kühle, Tanuja Chitnis, Brenda Banwell, Marc Tardieu, Douglas Arnold, Andrea Rawlings

Publikováno v: Revue Neurologique. 178:S117

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::9b5353800e0661474bb80bb8f2fda282
https://doi.org/10.1016/j.neurol.2022.02.393

Human SNORA31 variations impair cortical neuron-intrinsic immunity to HSV-1 and underlie herpes simplex encephalitis

Autor: Zobaida Alsum, Soraya Boucherit, Lluis Quintana-Murci, Peng Zhang, Luigi D. Notarangelo, Joseph A. Church, Daxing Gao, Rabih Halwani, Lazaro Lorenzo, Thomas M. Carlile, Maria F. Rojas-Duran, Ahmed Aziz Bousfiha, Yuval Itan, Laurent Abel, Flore Rozenberg, Fabien G. Lafaille, Wendy V. Gilbert, Jean-Laurent Casanova, Vimel Rattina, Marc Tessier-Lavigne, Lorenz Studer, Trine H. Mogensen, Bastian Zimmer, Benoit Henry, Søren R. Paludan, Shen-Ying Zhang, Franck Rapaport, Gregory A. Smith, Saleh Al-Muhsen, Michael J. Ciancanelli, Gaspard Kerner, Jessica L. McAlpine, Kerry Dobbs, Madalina E. Carter-Timofte, Laura Marques, Oliver Harschnitz, Osefame Ewaleifoh, Mary Hasek, Dominik Paquet, Marc Tardieu, Naima Amenzoui, Yoon Seung Lee, Dylan Kwart

Publikováno v: Lafaille, F G, Harschnitz, O, Lee, Y S, Zhang, P, Hasek, M L, Kerner, G, Itan, Y, Ewaleifoh, O, Rapaport, F, Carlile, T M, Carter-Timofte, M E, Paquet, D, Dobbs, K, Zimmer, B, Gao, D, Rojas-Duran, M F, Kwart, D, Rattina, V, Ciancanelli, M J, McAlpine, J L, Lorenzo, L, Boucherit, S, Rozenberg, F, Halwani, R, Henry, B, Amenzoui, N, Alsum, Z, Marques, L, Church, J A, Al-Muhsen, S, Tardieu, M, Bousfiha, A A, Paludan, S R, Mogensen, T H, Quintana-Murci, L, Tessier-Lavigne, M, Smith, G A, Notarangelo, L D, Studer, L, Gilbert, W, Abel, L, Casanova, J L & Zhang, S Y 2019, ' Human SNORA31 variations impair cortical neuron-intrinsic immunity to HSV-1 and underlie herpes simplex encephalitis ', Nature Medicine, vol. 25, no. 12, pp. 1873-1884 . https://doi.org/10.1038/s41591-019-0672-3
Nature Medicine
Nature Medicine, 2019, 25 (12), pp.1873-1884. ⟨10.1038/s41591-019-0672-3⟩
Nature Medicine, Nature Publishing Group, 2019, 25 (12), pp.1873-1884. ⟨10.1038/s41591-019-0672-3⟩
Nature medicine

International audience; Herpes simplex virus-1 (HSV-1) encephalitis (HSE) is typically sporadic. Inborn errors of TLR3- and DBR1-mediated central nervous system cell-intrinsic immunity can account for forebrain and brainstem HSE, respectively. We rep

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0dbe9bd2041435e0df6ada9573bd2856
https://pure.au.dk/portal/da/publications/human-snora31-variations-impair-cortical-neuronintrinsic-immunity-to-hsv1-and-underlie-herpes-simplex-encephalitis(81007800-1ad6-46e3-ab81-f589b19898cf).html

International Pediatric MS Study Group Global Members Symposium report: Table

Autor: Marc Tardieu, Daniela Pohl, Rogier Q. Hintzen, Lauren B. Krupp, Naila Makhani, Amy Waldman, Kevin Rostasy, Brenda Banwell, Emmanuelle Waubant, Evangeline Wassmer, Silvia Tenembaum, Tanuja Chitnis, Andrew J. Kornberg, Angelo Ghezzi, Maria Pia Amato

Publikováno v: Neurology, 87(9), S110-S116. Lippincott Williams & Wilkins

The International Pediatric Multiple Sclerosis Study Group held its inaugural educational program, "The World of Pediatric MS: A Global Update," in September 2014 to discuss advances and challenges in the diagnosis and management of pediatric multipl

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::87ec26dc9df856a0f8f90a15f88e6c25
https://doi.org/10.1212/wnl.0000000000002880