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Items: 4


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.


Oral Tolerance: Another Reason to Eat Your Veggies!

Hardet R, Mingozzi F.

Mol Ther. 2017 Feb 1;25(2):311-313. doi: 10.1016/j.ymthe.2017.01.001. Epub 2017 Jan 18. No abstract available.


Oral-tolerization Prevents Immune Responses and Improves Transgene Persistence Following Gene Transfer Mediated by Adeno-associated Viral Vector.

Hardet R, Chevalier B, Dupaty L, Naïmi Y, Riou G, Drouot L, Jean L, Salvetti A, Boyer O, Adriouch S.

Mol Ther. 2016 Feb;24(1):87-95. doi: 10.1038/mt.2015.146. Epub 2015 Aug 12.


Intrinsic transgene immunogenicity gears CD8(+) T-cell priming after rAAV-mediated muscle gene transfer.

Carpentier M, Lorain S, Chappert P, Lalfer M, Hardet R, Urbain D, Peccate C, Adriouch S, Garcia L, Davoust J, Gross DA.

Mol Ther. 2015 Apr;23(4):697-706. doi: 10.1038/mt.2014.235. Epub 2014 Dec 10.

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