Thibaut Marais
Pierre-and-Marie-Curie University
16 Papers
40 Citations
Thibaut Marais is an academic researcher from Pierre-and-Marie-Curie University. The author has contributed to research in topics: Biology & Gene delivery. The author has an hindex of 11, co-authored 14 publications. Previous affiliations of Thibaut Marais include French Institute of Health and Medical Research & Centre national de la recherche scientifique.
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Papers
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
S. Duque,Béatrice Joussemet,Christel Rivière,Thibaut Marais,Thibaut Marais,Laurence Dubreil,Anne Douar,John C. Fyfe,Philippe Moullier,Philippe Moullier,Marie Anne Colle,Martine Barkats,Martine Barkats +12 more
TL;DR: This is the first successful study of MN transduction in adult animals following intravenous (i.v.) delivery of self-complementary (sc) AAV9 vectors and this finding was successfully translated to large animals, with the demonstration of an efficient systemic scAAV9 gene delivery to the neonate and adult cat spinal cord.
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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
Elisa Dominguez,Thibaut Marais,Nicolas Chatauret,Nicolas Chatauret,Sofia Benkhelifa-Ziyyat,Sandra Duque,Philippe Ravassard,Philippe Ravassard,Philippe Ravassard,Romain Carcenac,Stéphanie Astord,Aurélie Pereira de Moura,Thomas Voit,Martine Barkats +13 more
TL;DR: This study reports the most efficient rescue of SMA mice to date after a single intravenous injection of an optimized SMN-encoding scAAV9, highlighting the considerable potential of this method for the treatment of human SMA.
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Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice.
Yannick Tanguy,Maria Grazia Biferi,Aurore Besse,Stéphanie Astord,Mathilde Cohen-Tannoudji,Thibaut Marais,Martine Barkats +6 more
TL;DR: It is found that AAVrh10 was more efficient than AAV9 for transduction of the dorsal spinal cord and the lower motor neurons (MNs), which suggest differences in the transduction mechanisms of these two serotypes, which both hold great promise for gene therapy of neurological diseases.
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Nicole Armbruster,Annalisa Lattanzi,Matthieu Jeavons,Laetitia van Wittenberghe,Bernard Gjata,Thibaut Marais,Samia Martin,Alban Vignaud,Thomas Voit,Fulvio Mavilio,Martine Barkats,Ana Buj-Bello +11 more
TL;DR: In this article, the authors reported a study aimed at analyzing the efficacy and biodistribution of a serotype-9, self-complementary AAV vector expressing a codon-optimized human SMN1 coding sequence under the control of the constitutive phosphoglycerate kinase (PGK) promoter in neonatal SMNΔ7 mice, a severe animal model of the disease.
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HLA-A*0201-restricted cytolytic responses to the rtTA transactivator dominant and cryptic epitopes compromise transgene expression induced by the tetracycline on system.
Florent Ginhoux,S. Turbant,David-Alexandre Gross,Jérôme Poupiot,Thibaut Marais,Yu Chun Lone,François A. Lemonnier,Hüseyin Firat,Norma Perez,Olivier Danos,Jean Davoust +10 more
TL;DR: The identification of HLA-A*0201 rtTA epitopes allowed us to demonstrate here that the delivery of the Tet-on system with weakly immunogenic rAAV vectors does not trigger primary CTL responses in mice, in contrast to DNA transfer.
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