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Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.

Guilbaud M, Devaux M, Couzinié C, Le Duff J, Toromanoff A, Vandamme C, Jaulin N, Gernoux G, Larcher T, Moullier P, Le Guiner C, Adjali O.

Hum Gene Ther. 2019 Jul;30(7):802-813. doi: 10.1089/hum.2018.234. Epub 2019 Apr 16.


Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls.

François A, Bouzelha M, Lecomte E, Broucque F, Penaud-Budloo M, Adjali O, Moullier P, Blouin V, Ayuso E.

Mol Ther Methods Clin Dev. 2018 Jul 27;10:223-236. doi: 10.1016/j.omtm.2018.07.004. eCollection 2018 Sep 21.


RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect.

Domenger C, Allais M, François V, Léger A, Lecomte E, Montus M, Servais L, Voit T, Moullier P, Audic Y, Le Guiner C.

Mol Ther Nucleic Acids. 2018 Mar 2;10:277-291. doi: 10.1016/j.omtn.2017.12.008. Epub 2017 Dec 21.


Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis.

Le Meur G, Lebranchu P, Billaud F, Adjali O, Schmitt S, Bézieau S, Péréon Y, Valabregue R, Ivan C, Darmon C, Moullier P, Rolling F, Weber M.

Mol Ther. 2018 Jan 3;26(1):256-268. doi: 10.1016/j.ymthe.2017.09.014. Epub 2017 Sep 19.


Long-term expression of melanopsin and channelrhodopsin causes no gross alterations in the dystrophic dog retina.

Ameline B, Tshilenge KT, Weber M, Biget M, Libeau L, Caplette R, Mendes-Madeira A, Provost N, Guihal C, Picaud S, Moullier P, Pichard V, Cronin T, Isiegas C.

Gene Ther. 2017 Nov;24(11):735-741. doi: 10.1038/gt.2017.63. Epub 2017 Sep 7.


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.


Accurate Identification and Quantification of DNA Species by Next-Generation Sequencing in Adeno-Associated Viral Vectors Produced in Insect Cells.

Penaud-Budloo M, Lecomte E, Guy-Duché A, Saleun S, Roulet A, Lopez-Roques C, Tournaire B, Cogné B, Léger A, Blouin V, Lindenbaum P, Moullier P, Ayuso E.

Hum Gene Ther Methods. 2017 Jun;28(3):148-162. doi: 10.1089/hgtb.2016.185. Epub 2017 Apr 21.


AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.

Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH.

Mol Ther. 2017 Jun 7;25(6):1375-1386. doi: 10.1016/j.ymthe.2017.04.001. Epub 2017 Apr 17.


Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates.

Tshilenge KT, Ameline B, Weber M, Mendes-Madeira A, Nedellec S, Biget M, Provost N, Libeau L, Blouin V, Deschamps JY, Le Meur G, Colle MA, Moullier P, Pichard V, Rolling F.

Hum Gene Ther Methods. 2016 Jun;27(3):122-34. doi: 10.1089/hgtb.2016.034.


Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR.

D'Costa S, Blouin V, Broucque F, Penaud-Budloo M, François A, Perez IC, Le Bec C, Moullier P, Snyder RO, Ayuso E.

Mol Ther Methods Clin Dev. 2016 Mar 30;5:16019. doi: 10.1038/mtm.2016.19. eCollection 2016.


AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs.

Pichard V, Provost N, Mendes-Madeira A, Libeau L, Hulin P, Tshilenge KT, Biget M, Ameline B, Deschamps JY, Weber M, Le Meur G, Colle MA, Moullier P, Rolling F.

Mol Ther. 2016 May;24(5):867-76. doi: 10.1038/mt.2016.37. Epub 2016 Feb 9.


Establishment of two quantitative nested qPCR assays targeting the human EPO transgene.

Neuberger EW, Perez I, Le Guiner C, Moser D, Ehlert T, Allais M, Moullier P, Simon P, Snyder RO.

Gene Ther. 2016 Apr;23(4):330-9. doi: 10.1038/gt.2016.2. Epub 2016 Jan 11.


Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing.

Lecomte E, Tournaire B, Cogné B, Dupont JB, Lindenbaum P, Martin-Fontaine M, Broucque F, Robin C, Hebben M, Merten OW, Blouin V, François A, Redon R, Moullier P, Léger A.

Mol Ther Nucleic Acids. 2015 Oct 27;4:e260. doi: 10.1038/mtna.2015.32.


Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype.

Servais L, Montus M, Guiner CL, Ben Yaou R, Annoussamy M, Moraux A, Hogrel JY, Seferian AM, Zehrouni K, Le Moing AG, Gidaro T, Vanhulle C, Laugel V, Butoianu N, Cuisset JM, Sabouraud P, Cances C, Klein A, Leturcq F, Moullier P, Voit T.

J Neuromuscul Dis. 2015 Sep 2;2(3):269-279.


Use of Adeno-Associated Virus to Enrich Cardiomyocytes Derived from Human Stem Cells.

Guan X, Wang Z, Czerniecki S, Mack D, François V, Blouin V, Moullier P, Childers MK.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):194-201. doi: 10.1089/humc.2015.052. Epub 2015 Aug 7.


Use of adeno-associated virus to enrich cardiomyocytes derived from human stem cells.

Guan X, Wang Z, Czerniecki S, Mack D, François V, Blouin V, Moullier P, Childers M.

Hum Gene Ther Clin Dev. 2015 Jul 15. [Epub ahead of print]


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.


Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.

Zerah M, Piguet F, Colle MA, Raoul S, Deschamps JY, Deniaud J, Gautier B, Toulgoat F, Bieche I, Laurendeau I, Sondhi D, Souweidane MM, Cartier-Lacave N, Moullier P, Crystal RG, Roujeau T, Sevin C, Aubourg P.

Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24. doi: 10.1089/humc.2014.139. Epub 2015 Apr 28.


Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats.

Hordeaux J, Dubreil L, Deniaud J, Iacobelli F, Moreau S, Ledevin M, Le Guiner C, Blouin V, Le Duff J, Mendes-Madeira A, Rolling F, Cherel Y, Moullier P, Colle MA.

Gene Ther. 2015 Apr;22(4):316-24. doi: 10.1038/gt.2014.121. Epub 2015 Jan 15.


Early interaction of adeno-associated virus serotype 8 vector with the host immune system following intramuscular delivery results in weak but detectable lymphocyte and dendritic cell transduction.

Gernoux G, Guilbaud M, Dubreil L, Larcher T, Babarit C, Ledevin M, Jaulin N, Planel P, Moullier P, Adjali O.

Hum Gene Ther. 2015 Jan;26(1):1-13. doi: 10.1089/hum.2014.070.


Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

Ayuso E, Blouin V, Lock M, McGorray S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, Giacca M, Kleinschmidt J, Leuchs B, Melas C, Mizukami H, Müller M, Noordman Y, Bockstael O, Ozawa K, Pythoud C, Sumaroka M, Surosky R, Tenenbaum L, van der Linden I, Weins B, Wright JF, Zhang X, Zentilin L, Bosch F, Snyder RO, Moullier P.

Hum Gene Ther. 2014 Nov;25(11):977-87. doi: 10.1089/hum.2014.057.


Transgene regulation using the tetracycline-inducible TetR-KRAB system after AAV-mediated gene transfer in rodents and nonhuman primates.

Le Guiner C, Stieger K, Toromanoff A, Guilbaud M, Mendes-Madeira A, Devaux M, Guigand L, Cherel Y, Moullier P, Rolling F, Adjali O.

PLoS One. 2014 Sep 23;9(9):e102538. doi: 10.1371/journal.pone.0102538. eCollection 2014.


Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Le Guiner C, Montus M, Servais L, Cherel Y, Francois V, Thibaud JL, Wary C, Matot B, Larcher T, Guigand L, Dutilleul M, Domenger C, Allais M, Beuvin M, Moraux A, Le Duff J, Devaux M, Jaulin N, Guilbaud M, Latournerie V, Veron P, Boutin S, Leborgne C, Desgue D, Deschamps JY, Moullec S, Fromes Y, Vulin A, Smith RH, Laroudie N, Barnay-Toutain F, Rivière C, Bucher S, Le TH, Delaunay N, Gasmi M, Kotin RM, Bonne G, Adjali O, Masurier C, Hogrel JY, Carlier P, Moullier P, Voit T.

Mol Ther. 2014 Nov;22(11):1923-35. doi: 10.1038/mt.2014.151. Epub 2014 Aug 4.


Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.

Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T.

Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23.


NGS library preparation may generate artifactual integration sites of AAV vectors.

Cogné B, Snyder R, Lindenbaum P, Dupont JB, Redon R, Moullier P, Leger A.

Nat Med. 2014 Jun;20(6):577-8. doi: 10.1038/nm.3578. No abstract available.


Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats.

Bucher T, Dubreil L, Colle MA, Maquigneau M, Deniaud J, Ledevin M, Moullier P, Joussemet B.

Gene Ther. 2014 May;21(5):522-8. doi: 10.1038/gt.2014.16. Epub 2014 Feb 27.


Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A.

Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523.


Different protein composition and functional properties of adeno-associated virus-6 vector manufactured from the culture medium and cell lysates.

Denard J, Jenny C, Blouin V, Moullier P, Svinartchouk F.

Mol Ther Methods Clin Dev. 2014 Jul 30;1:14031. doi: 10.1038/mtm.2014.31. eCollection 2014.


Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer.

Moreau A, Vandamme C, Segovia M, Devaux M, Guilbaud M, Tilly G, Jaulin N, Le Duff J, Cherel Y, Deschamps JY, Anegon I, Moullier P, Cuturi MC, Adjali O.

Mol Ther Methods Clin Dev. 2014 Jul 23;1:14028. doi: 10.1038/mtm.2014.28. eCollection 2014.


Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.

Lhériteau E, Petit L, Weber M, Le Meur G, Deschamps JY, Libeau L, Mendes-Madeira A, Guihal C, François A, Guyon R, Provost N, Lemoine F, Papal S, El-Amraoui A, Colle MA, Moullier P, Rolling F.

Mol Ther. 2014 Feb;22(2):265-277. doi: 10.1038/mt.2013.232. Epub 2013 Oct 4.


PCR-based detection of gene transfer vectors: application to gene doping surveillance.

Perez IC, Le Guiner C, Ni W, Lyles J, Moullier P, Snyder RO.

Anal Bioanal Chem. 2013 Dec;405(30):9641-53. doi: 10.1007/s00216-013-7264-8. Epub 2013 Aug 4. Review.


scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease.

Bucher T, Colle MA, Wakeling E, Dubreil L, Fyfe J, Briot-Nivard D, Maquigneau M, Raoul S, Cherel Y, Astord S, Duque S, Marais T, Voit T, Moullier P, Barkats M, Joussemet B.

Hum Gene Ther. 2013 Jul;24(7):670-82. doi: 10.1089/hum.2012.218.


Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy.

Petit L, Lhériteau E, Weber M, Le Meur G, Deschamps JY, Provost N, Mendes-Madeira A, Libeau L, Guihal C, Colle MA, Moullier P, Rolling F.

Mol Ther. 2012 Nov;20(11):2019-30. doi: 10.1038/mt.2012.134. Epub 2012 Jul 24.


Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors.

Veron P, Leborgne C, Monteilhet V, Boutin S, Martin S, Moullier P, Masurier C.

J Immunol. 2012 Jun 15;188(12):6418-24. doi: 10.4049/jimmunol.1200620. Epub 2012 May 16.


Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection.

Ni W, Le Guiner C, Moullier P, Snyder RO.

PLoS One. 2012;7(5):e36461. doi: 10.1371/journal.pone.0036461. Epub 2012 May 3.


Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6.

Denard J, Beley C, Kotin R, Lai-Kuen R, Blot S, Leh H, Asokan A, Samulski RJ, Moullier P, Voit T, Garcia L, Svinartchouk F.

J Virol. 2012 Jun;86(12):6620-31. doi: 10.1128/JVI.00297-12. Epub 2012 Apr 11.


Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver.

Nowrouzi A, Penaud-Budloo M, Kaeppel C, Appelt U, Le Guiner C, Moullier P, von Kalle C, Snyder RO, Schmidt M.

Mol Ther. 2012 Jun;20(6):1177-86. doi: 10.1038/mt.2012.47. Epub 2012 Mar 27.


Recombinant adeno-associated viral vector reference standards.

Moullier P, Snyder RO.

Methods Enzymol. 2012;507:297-311. doi: 10.1016/B978-0-12-386509-0.00015-6.


Failure of lower motor neuron radial outgrowth precedes retrograde degeneration in a feline model of spinal muscular atrophy.

Wakeling EN, Joussemet B, Costiou P, Fanuel D, Moullier P, Barkats M, Fyfe JC.

J Comp Neurol. 2012 Jun 1;520(8):1737-50. doi: 10.1002/cne.23010.


Biodistribution and shedding of AAV vectors.

Le Guiner C, Moullier P, Arruda VR.

Methods Mol Biol. 2011;807:339-59. doi: 10.1007/978-1-61779-370-7_15.


Neonatal systemic delivery of scAAV9 in rodents and large animals results in gene transfer to RPE cells in the retina.

Joussemet B, Belbellaa B, Mendes-Madeira A, Bucher T, Briot-Nivard D, Dubreil L, Colle MA, Cherel Y, Moullier P, Rolling F.

Exp Eye Res. 2011 Oct;93(4):491-502. doi: 10.1016/j.exer.2011.06.012. Epub 2011 Jun 24.


Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle.

Léger A, Le Guiner C, Nickerson ML, McGee Im K, Ferry N, Moullier P, Snyder RO, Penaud-Budloo M.

PLoS One. 2011;6(6):e20881. doi: 10.1371/journal.pone.0020881. Epub 2011 Jun 8.


A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.

Monteilhet V, Saheb S, Boutin S, Leborgne C, Veron P, Montus MF, Moullier P, Benveniste O, Masurier C.

Mol Ther. 2011 Nov;19(11):2084-91. doi: 10.1038/mt.2011.108. Epub 2011 May 31.


Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping.

Ni W, Le Guiner C, Gernoux G, Penaud-Budloo M, Moullier P, Snyder RO.

Gene Ther. 2011 Jul;18(7):709-18. doi: 10.1038/gt.2011.19. Epub 2011 Mar 10.


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

Ellinwood NM, Ausseil J, Desmaris N, Bigou S, Liu S, Jens JK, Snella EM, Mohammed EE, Thomson CB, Raoul S, Joussemet B, Roux F, Chérel Y, Lajat Y, Piraud M, Benchaouir R, Hermening S, Petry H, Froissart R, Tardieu M, Ciron C, Moullier P, Parkes J, Kline KL, Maire I, Vanier MT, Heard JM, Colle MA.

Mol Ther. 2011 Feb;19(2):251-9. doi: 10.1038/mt.2010.265. Epub 2010 Dec 7.


Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.

Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO.

Hum Gene Ther. 2010 Oct;21(10):1273-85. doi: 10.1089/hum.2009.223.


Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors.

Lhériteau E, Libeau L, Mendes-Madeira A, Deschamps JY, Weber M, Le Meur G, Provost N, Guihal C, Moullier P, Rolling F.

Mol Ther. 2010 Jun;18(6):1085-93. doi: 10.1038/mt.2010.46. Epub 2010 Mar 30.


Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle.

Toromanoff A, Adjali O, Larcher T, Hill M, Guigand L, Chenuaud P, Deschamps JY, Gauthier O, Blancho G, Vanhove B, Rolling F, Chérel Y, Moullier P, Anegon I, Le Guiner C.

Mol Ther. 2010 Jan;18(1):151-60. doi: 10.1038/mt.2009.251. Epub 2009 Nov 3.


Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.

Colle MA, Piguet F, Bertrand L, Raoul S, Bieche I, Dubreil L, Sloothaak D, Bouquet C, Moullier P, Aubourg P, Cherel Y, Cartier N, Sevin C.

Hum Mol Genet. 2010 Jan 1;19(1):147-58. doi: 10.1093/hmg/ddp475.


Adeno-associated virus capsid serotype identification: Analytical methods development and application.

Van Vliet K, Mohiuddin Y, McClung S, Blouin V, Rolling F, Moullier P, Agbandje-McKenna M, Snyder RO.

J Virol Methods. 2009 Aug;159(2):167-77. doi: 10.1016/j.jviromet.2009.03.020. Epub 2009 Mar 26.


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