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Items: 24


Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cédrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, Galy A.

Mol Ther Methods Clin Dev. 2019 Sep 13;15:232-245. doi: 10.1016/j.omtm.2019.08.014. eCollection 2019 Dec 13.


Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.

Da Rocha S, Bigot J, Onodi F, Cosette J, Corre G, Poupiot J, Fenard D, Gjata B, Galy A, Neildez-Nguyen TMA.

Mol Ther Methods Clin Dev. 2019 Jul 23;14:285-299. doi: 10.1016/j.omtm.2019.06.011. eCollection 2019 Sep 13.


An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.

Israeli D, Cosette J, Corre G, Amor F, Poupiot J, Stockholm D, Montus M, Gjata B, Richard I.

Mol Ther Methods Clin Dev. 2019 May 10;13:494-502. doi: 10.1016/j.omtm.2019.04.007. eCollection 2019 Jun 14.


AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

Colella P, Sellier P, Costa Verdera H, Puzzo F, van Wittenberghe L, Guerchet N, Daniele N, Gjata B, Marmier S, Charles S, Simon Sola M, Ragone I, Leborgne C, Collaud F, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Nov 17;12:85-101. doi: 10.1016/j.omtm.2018.11.002. eCollection 2019 Mar 15.


Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269. doi: 10.1016/j.omtm.2018.03.002. eCollection 2018 Jun 15.


Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F.

Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28.


Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.


Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G.

Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105.


A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F.

Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. eCollection 2016.


Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A.

Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016.


Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA.

Dupont JB, Tournaire B, Georger C, Marolleau B, Jeanson-Leh L, Ledevin M, Lindenbaum P, Lecomte E, Cogné B, Dubreil L, Larcher T, Gjata B, Van Wittenberghe L, Le Guiner C, Penaud-Budloo M, Snyder RO, Moullier P, Léger A.

Mol Ther Methods Clin Dev. 2015 Apr 8;2:15010. doi: 10.1038/mtm.2015.10. eCollection 2015.


Longitudinal in vivo muscle function analysis of the DMSXL mouse model of myotonic dystrophy type 1.

Decostre V, Vignaud A, Matot B, Huguet A, Ledoux I, Bertil E, Gjata B, Carlier PG, Gourdon G, Hogrel JY.

Neuromuscul Disord. 2013 Dec;23(12):1016-25. doi: 10.1016/j.nmd.2013.07.014. Epub 2013 Aug 12.


A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C.

Herson S, Hentati F, Rigolet A, Behin A, Romero NB, Leturcq F, Laforêt P, Maisonobe T, Amouri R, Haddad H, Audit M, Montus M, Masurier C, Gjata B, Georger C, Cheraï M, Carlier P, Hogrel JY, Herson A, Allenbach Y, Lemoine FM, Klatzmann D, Sweeney HL, Mulligan RC, Eymard B, Caizergues D, Voït T, Benveniste O.

Brain. 2012 Feb;135(Pt 2):483-92. doi: 10.1093/brain/awr342. Epub 2012 Jan 11.


Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models".

Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A.

Mol Ther. 2009 Jul;17(7):1300. doi: 10.1038/mt.2009.61. Epub 2016 Dec 6. No abstract available.


Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.

Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M, Gjata B, Montus M, Siminovitch K, Aiuti A, Naldini L, Dupré L, Roncarolo MG, Galy A, Villa A.

Mol Ther. 2009 Jun;17(6):1073-82. doi: 10.1038/mt.2009.31. Epub 2009 Mar 3. Erratum in: Mol Ther. 2009 Jul;17(7):1300.


Analysis of growth factor expression in affected and unaffected muscles of oculo-pharyngeal muscular dystrophy (OPMD) patients: a pilot study.

Bouazza B, Kratassiouk G, Gjata B, Perie S, Lacau St Guily J, Butler-Browne GS, Svinartchouk F.

Neuromuscul Disord. 2009 Mar;19(3):199-206. doi: 10.1016/j.nmd.2008.12.003. Epub 2009 Jan 29.


Expression of mdr1 is required for efficient long term regeneration of dystrophic muscle.

Israeli D, Ziaei S, Gjata B, Benchaouir R, Rameau P, Marais T, Fukada S, Segawa M, Yamamoto H, Gonin P, Danos O, Garcia L.

Exp Cell Res. 2007 Jul 1;313(11):2438-50. Epub 2007 Apr 1.


CD4+CD25+ regulatory T cells inhibit immune-mediated transgene rejection.

Gross DA, Leboeuf M, Gjata B, Danos O, Davoust J.

Blood. 2003 Dec 15;102(13):4326-8. Epub 2003 Jul 31.


Regulatory function of in vivo anergized CD4(+) T cells.

Jooss K, Gjata B, Danos O, von Boehmer H, Sarukhan A.

Proc Natl Acad Sci U S A. 2001 Jul 17;98(15):8738-43. Epub 2001 Jul 3.


Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors.

Sarukhan A, Camugli S, Gjata B, von Boehmer H, Danos O, Jooss K.

J Virol. 2001 Jan;75(1):269-77.


Sex and parity modulate cytokine production during murine ageing.

Barrat F, Lesourd B, Boulouis HJ, Thibault D, Vincent-Naulleau S, Gjata B, Louise A, Neway T, Pilet C.

Clin Exp Immunol. 1997 Sep;109(3):562-8.


Factors inducing mast cell accumulation in skeletal muscle.

Lefaucheur JP, Gjata B, Sebille A.

Neuropathol Appl Neurobiol. 1996 Jun;22(3):248-55.


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