Scientific publications before 2014
2014
NGS Library preparation may generate artifactual intégration sites of AAV vectors.
Cogne B, Snyder R, Lindenbaum P, Dupont JB, Redon R, Moullier P, Léger A.
Nat Med 2014 June 5 ;20(6) :577-8.
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, Riviere C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Gua, 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)
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.May ;21(5) :522-8
In the rat liver, adenoviral gene transfer efficiency is comparable to AAV.
Montenegro-Miranda PS, Pichard V, Aubert D, Ten Bloemendaal L, Duijst S, de Waart DR, Ferry N, Bosma PJ.
Gene Ther.2014 Feb ;21(2) :168-74.Epub2013 Nov 28.
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) :265-77.Epub 2013 Oct4.
Immuno-Histochemical analysis of rod and cone reaction to RPE65 deficiency in the inferior and superior canine retina.
Klein D, Mendes-Madeira A, Schlegel P, Rolling F, Lorenz B, Haverkamp S, Stieger K.
PLoS One. 2014 Jan 21;9(1):e86304. doi: 10.1371/journal.pone.0086304. eCollection 2014 Jan 21
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.
Generation and in vivo evaluation of IL10-treated tolerogenic dendritic cells in a nonhuman primate model of AAV-based gene transfer.
Moreau A, Vandamme C, Segovia M, Marie Devaux, 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.
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.
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 Aug 4. doi: 10.1038/mt.2014.151. [Epub ahead of print]
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.
Characterization of dystrophin deficient rats : a new model for Duchenne muscular dystrophy.
Larcher T, Lafoux A, Tesson L, Remy S, Thepenier V, François V, Le Guiner C, Goubin H, Dutilleul M, Guigand L, Toumaniantz G, De Cian A, Boix C, Renaud JB, Cherel Y, Giovannangeli C, Condorcet JP, Anegon I, Huchet C.
PLoS One.2014 Oct 13 ;9(10) :e110371. Doi : 10.1371/journal.pone.01100371. eCollection 2014.
Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients.
Jeanson-Leh L, MLameth J, Krimi S, Buisset J, Amor F, Le Guiner C, Barthélémy I, Servais L, Blot S, Voit T, Israeli D.
Am J Pathol. 2014 Sep 3. S0002-9440(14)00439-8 Epub ahead of print.
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, and Svinartchouk F.
Molecular Therapy – Methods & Clinical Development. July 2014.
2013
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, Wakelin 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. Jun 25 (Epub ahead of print).
Mutations in FAM111B cause hereditary fibrosing poikiloderma with tendon contracture, myopathy, and pulmonary fibrosis.
Mercier S, Küry S, Shaboodien G, Houniet DT, Khumalo NP, Bou-Hanna C, Bodak N, Cormier-Daire V, David A, Faivre L, Figarella-Branger D, Gherardi RK, Glen E, Hamel A, Laboisse C, Le Caignec C, Lindenbaum P, Magot A, Munnich A, Mussini JM, Pillay K, Rahman T, Redon R, Salort-Campana E, Santibanez-Koref M, Thauvin C, Barbarot S, Keavney B, Bézieau S, Mayosi BM.
Am J Hum Genet. Dec 5
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
2012
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
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 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, Kalle CV, Snyder RO, Schmidt M.
Mol Ther.2012 Mar 27
Recombinant adeno-associated viral vector reference standards.
Moullier P, Snyder RO.
Methods Enzymol.2012 ;507 :297-311
Failure of lower motor neuron radial outgrowth precedes retrograde degeneration in a feline model of SMA.
Wakeling EN, Joussemet B, Costiou P, Fanuel D, Moullier P, Barkats M, Fyfe LC.
J Comp Neurol. 2012 Jun 1 ;520(8) :1737-1750
Restoration of vision in the PDE6B-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
Gene transfer of human CD40Ig does not prevent rejection in a non-human primate kidney allotransplantation model.
Angin M, Poirier N, Dilek N, Le Guiner C, Toromanoff A, Blancher A, Cherel Y, Deschamps JY, Tillou X, Renaudin K, Minault D, Hervouet J, Blancho G, Vanhove B, Anegon I, Le Mauff B.
Transpl Immunol. 2012 Dec;27(4):139-45.
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 Jun15 ;188(12) :6418-24
MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV.
Sudres M, Ciré S, Vasseur V, Brault L, Da Rocha S, Boisgérault F, Le Bec C, Gross DA, Blouin V, Ryffel B, Galy A.
Mol Ther. 2012 Aug
2011
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.Epub 2011 Mar 10.
Biodistribution and shedding of AAV vectors.
Le Guiner C, Moullier P, Arruda VR.
Methods Mol Biol. 2011 ;807 :339-59
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.Epub 2011 Jun 24.
Adeno-associated viral vector-mediated transgene expression is independant 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.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.
Montheilhet 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
Matrix metalloproteinase 14 overexpression reduces corneal scarring.
Galiacy SD, Fournié P, Massoudi D, Ancèle E, Quintyn JC, Erraud A, Raymind-Letron I, Rolling F, Malecaze F.
Gene Ther.2011 May ;18(5) :462-8
Adeno-associated virus mediated gene therapy for retinal degenerative diseases.
Stieger K, Cronin T, Bennett J, Rolling F.
Methods Mol Biol.2011 ;807 :179-218
Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in Duchenne muscular dystrophy dogs.
Rouger K, Larcher T, Dubreil L, Deschamps JY, Le Guiner C, Jouvion G, Delorme B, Lieubeau B, Carlus M, Fornasari B, Theret M, Orlando P, Ledevin M, Zuber C, Leroux I, Deleau S, Guigand L, Testault I, Le Rumeur E, Fiszman M, Chérel Y.
Am J Pathol. 2011 Nov ;179(5) :2501-18
Measuring immune responses to recombinant AAV gene transfer.
Martino AT, Herzog RW, Anegon I, Adjali O.
Methods Mol Biol. 2011 ;807 :259-72
Methods in Molecular Biology 807, 2011 – Adeno-Associated Virus : Methods and Protocols
Richard O.Snyder, Philippe Moullier Editors.
Book
Evaluation of the fate of rAAV genomes following in vivo administration.
Clark KR, Penaud-Budloo M.
Methods Mol Biol. 2011 ;807 :239-258.
Production and purification of recombinant adeno-associated vectors.
Wang L, Blouin V, Brument N, Bello-Roufai M, Francois A.
Methods Mol Biol. 2011 ;807 :361-404.
2010
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 18(1) : 151-160
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. 19(1) :147-158
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.Epub 2010 Mar 30.
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.
Human Gene Therapy, 2010 Oct;21(10):1273-85.
Preclinical studies on specific gene therapy for recessive retinal degenerative diseases.
Stieger K, Chauveau C, Rolling F.
Cur Gene Ther.2010 Oct ;10(5) :389-403
AAV mediated gene therapy for the treatment of retinal diseases.
Rolling F.
Curr Gene Ther.2010 Oct
2009
1. Efficient intrathymic gene transfer following in situ administration of a rAAV serotype 8 vector in mice and nonhuman primates.
Moreau A, Vicente R, Dubreuil L, Adjali O, Podevin G, Jacquet C, Deschamps JY, Klatzmann D, Cherel Y, Taylor N, Moullier P, Zimmermann VS,
Mol. Ther. 2009 17(3):472-9
2. Detection of Intact rAAV Particles up to 6 Years After Successful Gene Transfer in the Retina of Dogs and Primates.
Stieger K, Schroeder J, Provost N, Mendes-Madeira A, Belbellaa B, Meur GL, Weber M, Deschamps JY, Lorenz B, Moullier P, Rolling F.
Mol Ther. 2009 17(3):516-23.
3. AAV-mediated gene therapy for retinal disorders in large animal models.
Stieger K, Lheriteau E, Moullier P, Rolling F.
ILAR J. 2009;50(2):206-24.
4. The RPGRIP1-deficient dog, a promising canine model for gene therapy
Lhériteau E, Libeau L, Stieger K, Deschamps JY, Mendes-Madeira A, Provost N, Lemoine F, Mellersh C, Ellinwood NM, Cherel Y, Moullier P, Rolling F.
Mol Vis. 2009;15:349-61.
5. 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. Epub 2009 Mar 26.
6. Tolerogenic dendritic cells actively inhibit T cells through heme oxygenase-1 in rodents and in nonhuman primates.
Moreau A, Hill M, Thébault P, Deschamps JY, Chiffoleau E, Chauveau C, Moullier P, Anegon I, Alliot Licht B, Cuturi MC.
FASEB J. 2009 Sep;23(9):3070-7. Epub 2009 May 6.
7. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.
Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M.
Mol Ther. 2009 Jul;17(7):1187-96. Epub 2009 Apr 14.
8. Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution.
Ciron C, Cressant A, Roux F, Raoul S, Cherel Y, Hantraye P, Déglon N, Schwartz B, Barkats M, Heard JM, Tardieu M, Moullier P, Colle MA.
Hum Gene Ther. 2009 Apr;20(4):350-60.
9. In vivo gene regulation using tetracycline-regulatable system.
Stieger K, Belbellaa B, Le Guiner C, Moullier P, Rolling F.
Advanced Drug Delivery Reviews. 2009 2-61(7-8):527-41.
2008
1. Relative influence of the adeno-associated virus (AAV) type 2 p5 element for recombinant AAV vector site-specific integration.
Guilbaud M, Chadeuf G, Avolio F, François A, Moullier P, Recchia A, Salvetti A.
J Virol. 2008 Mar;82(5):2590-3.
2. International efforts for recombinant Adeno-Associated viral vector reference standards.
Moullier P, Snyder RO.
Mol. Ther 2008 Jul ;16(7) :1185-8
3. Subretinal Delivery of Recombinant AAV Serotype 8 Vector in Dogs Results in Gene Transfer to Neurons in the Brain.
Stieger K, Colle MA, Dubreil L, Mendes-Madeira A, Weber M, Le Meur G, Deschamps JY, Provost N, Nivard D, Cherel Y, Moullier P, Rolling F.
Mol Ther. 2008 May ;16(5) :916-23.Epub 2008 Mar 11
4. Safety and efficacy of regional intravenous (RI) versus intramuscular (IM) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.
Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.
Mol Ther. 2008 Jul ;16(7) :1291-9.Epub 2008 May 6
5. Superiority of bon marrow-derived dendritic cells over monocyte-derived ones for the expansion of regulatory T cells in the macaque.
Moreau A, Chiffoleau E, Beriou G, Deschamps JY, Heslan M, Ashton-Chess J, Rolling F, Josien R, Moullier P, Cuturi MC, Alliot-Licht B.
Transplantation 2008 May 15 ;85(9) :1351-1356
6. Adeno-associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle.
Penaud-Budloo M, Le Guiner C, Nowrouzi A, Toromanoff A, Chérel Y, Chenuaud P, Schmidt M, Von Kalle C, Rolling F, Moullier P, Snyder RO.
J Virol. 2008 Aug ;82(16) :7875-85.Epub2008 Jun4).
7. The role of the adeno-associated virus capsid in gene transfer.
Van Vliet KM, Blouin V, Brument N, Agbandje-McKenna M, Snyder RO.
Methods Mol Biol, 2008 ;437 :51-91.
2007
1. Stable producer cell lines for adeno-associated virus (AAV) assembly.
Chadeuf G, Salvetti A.
Cold Spring Harb Protoc. 2010 Oct 1;2010(10):pdb.prot5496. doi: 10.1101/pdb.prot5496.
2. Restoration of vision in RPE65 deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
Le Meur G, Stieger K, Smith, AJ ; Weber M, Deschamps, J-Y, Nivard D, Mendes-Madeira A, Provost N, Péréon Y, Chérel Y, Ali RR, Hamel C, Moullier P, Rolling F. .
Gene Ther.2007 Feb ;14(4) :292-303. Epub 2006
3. Immune responses to Gene Product of inducible Promoters.
Le Guiner C, Stieger K, Snyder R, Rolling F, Moullier P .
Current Gene Therapy.2007 Oct ;7(5) :334-46
4. Oral administration of doxycycline allows tight control of transgene expression : a key step towards gene therapy of retinal diseases.
Stieger K, Mendes-Madeira A, Meur GL, Weber M, Deschamps JY, Nivard D, Provost N, Moullier P, Rolling F.
Gene Ther 2007 Dec ;14(23) :1668-73.Epub 2007 Oct 4.
2006
1. Impact of the interaction between Herpes Simplex Virus type 1 regulatory ICPO and ubiquitin specific protease USP7 on the activation on adeno-associated Virus type 2 rep gene expression.
Geoffroy M.C, Chadeuf G, Orr A, Salvetti A and Everett R.
J Virol 80(7) :3650-4
2. Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors.
Stieger K, Le Meur G, Lasne F, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Martin L, Moullier P, Rolling F.
Mol Ther13(5) :967-75
3. Gene Therapy of the brain in the dog model of Hurler’s syndrome.
Ciron C, Desmaris N, Colle MA, Raoul S, Joussemet B, Verot L, Ausseil J, Froissart R, Roux F, Cherel Y, Ferry N, Lajat Y, Schwartz B, Vannier MT, Maire I, Tarideu M, Moullier P, Heard JM.
Ann Neurol.2006 Aug ;60(2) :204-13
4. Proteolytic Mapping of the adeno-associated virus capsid.
Kim Van Vliet and Veronique Blouin, Mavis Agbandje-McKenna, Richard O.Snyder.
Mol Ther.2006 dec ;14(6) :809-21
5. Gene therapeutic prospects in early onset of severe retinal dystrophy : restoration of vision in RPE65 Briard dogs using an AAv serotype 4 vector that specifically targets the retinal pigmented epithelium.
Rolling F, Le Meur G, Stieger K, Smith AJ, Weber M, Deshamps JY, Nivard D, Mendes- Madeira A, Provost N, Pereon Y, Cherel Y, Ali RR, Hamel C, Moullier P.
Bull Mem Acad R Med Belg. 2006 ;161(10-12) :497-508
2005
1. Biodistribution of rAAV vectors following intraocular administration : evidence for the presence and persistence of vector DNA in the optic nerve and in the brain.
Provost N, Le Meur G, Weber M, Mendes-Madeira A, Podevin G, Cherel Y, Colle MA, Deschamps JY, Moullier P, Rolling F.
Mol Ther 11(2):275-83.
2. Postsurgical assessment and long term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates.
Le Meur G, Weber M, Pereon Y, Mendes-Madeira A, Nivard D, Deschamps JY, Moullier M, Rolling F.
Arch. Ophthalmol. 123:500-506.
3. Intracellular route and biological activity of exogenously delivered Rep proteins from the adeno-associated virus.
Awedikian R, François A, Guilbaud M, Moullier P, Salvetti A.
Virology. 335(2) :252-263.
4. Evidence for encapsidation of prokaryotic sequences during rAAV production and their in vivo persistence after vector delivery.
Chadeuf G, Ciron C, Moullier P, Salvetti A.
Mol. Ther. Oct ;12(4) :744-53.
5. In vivo gene transfer to the rat retina using herpes simplex virus type 1 (HSV-1)-based amplicon vectors.
Fraefel C, Mendes-Madeira A, Mabon O, Lefevre A, Le Meur G, Ackermann M, Moullier P, Rolling F.
Gene Ther. 12(16):1283-1288
6. The cellular TATA binding protein is required for Rep-dependent replication of a minimal adeno-associated virus type 2 p5 element.
Francois A, Guilbaud M, Awedikian R, Chadeuf G, Moullier P, Salvetti A.
J. Virol. 79(17):11082-11094
7.Adeno-associated viral vectors for retinal gene transfer and treatment of retinal diseases.
Auricchio A, Rolling F.
Curr Gene Ther 5(3) :339-48
8. Helper functions required for wild type and recombinant adeno-associated virus growth.
Geoffroy M.C, Salvetti A.
Current Gene Therapy 12(4) : 744-53
9. The Twelfth annual meeting of European Society of Gene Therapy.
Grez M, Galun E, Moullier P.
Mol Ther. 2005 Feb ;11(2) :178-9
10. Gene therapy of the brain in the dog model of Hurler's syndrome.
Ciron C, Desmaris N, Colle MA, Raoul S, Joussemet B, Vérot L, Ausseil J, Froissart R, Roux F, Chérel Y, Ferry N, Lajat Y, Schwartz B, Vanier MT, Maire I, Tardieu M, Moullier P, Heard JM.
Ann Neurol. 2006 Aug;60(2):204-13.
11. Tetracycline-inducible viral interleukin-10 intraocular gene transfer, using adeno-associated virus experimental autoimmune uveoretinitis.
Smith JR, Verwaerde C, Rolling F, Naud MC, Delanove A, Thillaye-Goldenberg B, Apparailly F, de Kozak Y.
Hum Gene Ther. 16(9) :1037-1046.
2004
1. Autoimmune anemia in macaques following erythropoietin gene therapy.
Chenuaud P , Larchet T, Rabinowitz JE, Provost N, Cherel Y, Casadevall N, Samulski RJS, Moullier P.
Blood. 103(9) :3303-3304.
2. Optimal design of a single recombinant adeno-associated virus derived from serotypes 1 and 2 to achieve more tightly regulated transgene expression from nonhuman primate muscle.
Chenuaud P , Larcher T, Rabinowitz JE, Provost N, Joussemet B, Bujard H, Samulski RJS, Favre D, Moullier P.
Mol. Ther. 9 :410-418
3. Factors influencing immune response after in vivo retrovirus-mediated gene transfer to the liver.
Podevin G, Otta E ;, Nguyen JM, Pichard V, Aubert D, Moullier P, Ferry N.
J Gene Med. 6(1) :16-21
4. Identification of a replication-defective herpes simplex virus for recombinant adeno-associated virus type 2 (rAAV2) particle assembly using stable producer cell lines.
Toublanc E, Benraiss A, Bonnin D, Blouin V, Brument N, Cartier N, Epstein AL, Moullier P, Salvetti A.
J. Gene Med. 6(5) :555-564
5. Improving rAAV production and purification : towards the definition of a scaleable process.
Blouin V, Brument N, Toublanc E, Raimbaud I, Moullier P, Salvetti A.
J Gene Med. 2004. 6:S223-S228.
6. Early detection of a two-long-terminal-repeat junction molecule in the cytoplasm of recombinant murine leukemia virus-infected cells.
Serhan F, Penaud M, Petit C, Leste-Lasserre T,Trajcevski S, Klatzmann D. Duisit G, Sonigo P, Moullier P.
J. Virol. 78(12) :6190-6199.
7. The herpes simplex virus type 1 ICP0 protein mediates the activation of adeno-associated virus type 2 rep gene expression from a latent integrated form.
Geoffroy MC, Epstein AL, Toublanc E, Moullier P, Salvetti A.
J. Virol. 78(20) :10977-10986.
8. Recombinant AAV-mediated gene transfer to the retina : gene therapy perspectives.
Rolling F.
Gene Ther.11 :S26-S32.
9.« Genetic doping” with erythropoietin cDNA in primate muscle is detectable.
Lasne F, Martin L, de Ceaurriz J, Larcher T, Moullier P, Chenuaud P.
Mol. Ther. 10(3) 409-410.
10. AAV gene transfer to the retina does not protect retrovirally transduced hepatocytes from the immune response.
Bellodi-Privato M, Le Meur G, Aubert D, Mendes-Madera A, Pichard V, Rolling F, Ferry N.
J Mol Med.;82(6):403-10.
11. La Génétique.
Fabre-Magnan M, Moullier P.
Science Humaine. Collection « Débats ». Editions Belin. Avril 2004
2003
1. Sustained tetracyline-regulated transgene expression in vivo in rat retinal ganglion cells using a type 2 adeno-associated viral vector.
Folliot S, Briot D, Conrath H, Provost N, Cherel Y, Moullier P, Rolling F.
J. Gene Med. 5(6) :493-501.
2. Evidence for packaging rep-cap sequences into Adeno-Associated Virus (AAV) type 2 capsids in the absence of the Inverted Terminal Repeats: a model for the generation of rep-positive AAV particles.
Nony P, Chadeuf G, Tessier J, Moullier P, Salvetti A.
J. Virol 77(1) :776-781.
3. Recombinant Adeno-Associated Virus serotype 4 mediates unique and exclusive long term transduction of retinal pigmented epithelium in rat, dog and nonhuman primate after subretinal delivery.
Weber M, Rabinowitz J, Provost N, Conrath H, Folliot S, Briot D, Chérel Y, Chenuaud P, Samulski J, Moullier P. and Rolling, F,
Mol. Ther 7(6) :774-781.
4. Cytotoxic immune response after retroviral-mediated hepatic gene transfer in rat does not preclude expression from adeno-associated virus 1 transduced muscles.
Aubert D, Pichard V, Ferry N.
Hum. Gene Ther.14(5) :473-481
2002
1. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity.
Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X, Samulski RJS.
J. Virol 76 :791-801
2. Cytotoxic immune response blunts long term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat.
Aubert D, Ménoret S, Chiari E, Pichard V, Durand S, Tesson L, Moullier P, Anegon I, Ferry N.
Mol. Ther. 5:388-396.
3.Recombinant adeno-associated virus type 2 mediates highly efficient gene transfer in regenerating rat skeletal muscle.
Abadie J, Blouin V, Guigand L, Wyers M, Cherel Y.
Gene Ther. 9(15):1037-43.
4. Five recombinant simian immunodeficiency virus (SIV) pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat.
Duisit G, Conrath H, Saleun S, Folliot S, Provost N, Cosset F.-L, Sandrin V, Moullier P, Rolling F..
Mol. Ther. 6(4) :446-454
5. Characterization of a Producer Cell-Dependent Restriction of MLV Replication.
Serhan F, Jourdan N, Saleun S, Moullier P, Duisit G.
J. Virol. 76:6609-6617.
6. Liver regeneration: with help from marrow.
Ferry N, Hadchouel M.
J. Hepatol. 36:695-697.
7. Lack of immune response against the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated transgene expression in nonhuman primates after intramuscular injection of recombinant adeno-associated virus.
Favre D, Blouin V, Provost N, Spisek R, Porrot F, Bohl D, Marmé F, Chérel Y, Salvetti A, Hurtrel B, Heard J.-M, Rivière Y, Moullier P.
J. Virol. 76(22) 11605-11611
8. A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes –2 and –5.
Brument N, Morenweiser R, Blouin V, Toublanc E, Raimbaud I, Chérel Y, Folliot S, Gaden F, Boulanger P, Kroner-Lux G, Moullier P, Rolling F, Salvetti A.
Mol. Ther. 6:678-686.
9. Differential sensitivity of endothelial cells of various species to apoptosis induced by gene transfer of Fas ligand: role of FLIP levels.
Bouchet D, Tesson L, Ménoret S, Charreau B, Mathieu P, Yagita H, Duisit G, Anegon I.
Mol. Med. 8(10) ;612-623
10. In vivo cell lineage analysis during chemical hepatocarcinogenesis in rats using retroviral mediated gene transfer : Evidence for dedifferentiation of mature hepatocytes.
Gournay J, Auvigne I, Pichard V, Ligeza C, Bralet M.-P, Ferry N.
Lab. Invest 82(6):781-8.
11. LacZ transgenic rats tolerant for beta-galactosidase: recipients for gene transfer studies using lacZ as reporter gene.
Menoret S, Aubert D, Tesson L, Braudeau C, Pichard V, Ferry N, Anegon I.
Hum. Gene Ther. 13(11):1383-1390.
12. Demonstration of direct lineage between hepatocytes and hepatocellular carcinoma in diethylnitrosamine-treated rats.
Bralet MP, Pichard V, Ferry N.
Hepatology 36(3):623-630.
13. Lentiviral vectors derived from simian immunodeficiency virus.
Negre D, Duisit G, Mangeot PE, Moullier P, Darlix JL, Cosset FL.
Curr Top Microbiol Immunol. 261:53-74
14. In vivo study of mitogenic and tumor initiating effects of cyproterone acetate using cell lineage analysis of genetically labeled hepatocytes.
Auvigne I, Pichard V, Aubert D, Robillard N, Ferry N.
Hepatology . 35(2):281-288
2001
1. The immune-dependent distant bystander effect after adenovirus-mediated suicide gene transfer in a rat model of liver colorectal metastasis Cancer.
Ligeza C, Agard C, Dupas B, Izembart A, El Kouri C, Moullier P, Ferry N.
Gene Ther. 8(2) : 128-136.
2. Characterization of adenovirus-induced inverted terminal repeat-independent amplification of integrated adeno-associated virus rep-cap sequences.
Tessier J, Chadeuf G, Nony P, Avet-Loiseau H, Moullier P, Salvetti A.
J. Virol. 75(1) 375-383
3. Novel cis-acting element in the Adeno-Associated type 2 genome involved in amplification of integrated rep-cap sequences.
Nony P, Tessier J, Chadeuf G, Ward P, Giraud A, Dugast M, Linden R.M, Moullier P, Salvetti A.
J. Virol 75 : 9991-9994
4. Primary adult human astrocytes as an ex vivo vehicle for ß-glucuronidase delivery in the brain.
Serguera C, Sarkis C, Ridet JL, Colin P, Moullier P, Mallet J.
Mol. Ther 3(6) :875-881
5. Efficient retroviral gene transfer to the liver in vivo using nonpolypeptidic mitogens.
Pichard V, Aubert D, Ferry N.
Biochem. Biophys. Res. Comm. 286(5):929-935
6. Immediate and long-term safety of recombinant Adeno-Associated Virus (r-AAV) injection into the nonhuman primate muscle.
Favre D, Provost N, Blouin V, Blancho G, Chérel Y, Salvetti A, Moullier P.
Mol. Ther. 4(6):559-566
7. Adenoviral vector-mediated beta-glucuronidase cDNA transfer to treat MPS VII RPE in vitro.
Verdugo ME, Scarpino V, Moullier P, Haskins ME, Aguirre GD, Ray J.
Curr Eye Res. 23(5) :357-367.
2000
1. In vivo retrovirus-mediated gene transfer into lamb liver.
Podevin G, Podevin J, Ongoiba N, Sandoval C, Bralet MP, Ferry N, Levard G.
Eur. J. Pediatr. Surg. 10(3) : 167-171
2. Efficient recombinant adeno-associated virus production by a stable rep-cap HeLa cell line correlates with adenovirus-induced amplification of the integrated rep-cap genome.
Chadeuf G, Favre D, Tessier J, Provost N, Nony P, Kleinschmidt J, Moullier P, Salvetti A.
J. Gene Med. 2 :260-268
3. Hyaluronidase enhances recombinant Adeno-Associated Virus(rAAV)-mediated gene transfer in the rat skeletal muscle
Favre D, Chérel Y, Provost N, Blouin V, Ferry N, Moullier P, Salvetti A. .
Gene Ther. 7 : 1417-1420
4. Critical aspects of viral vectors for gene transfer into the kidney.
Favre D, Ferry N, Moullier P.
J. Am. Soc. Neph 11 :S149-S153
5. Improvement of erythropoiesis in ß-thalassemic mice by continuous erthropoietin delivery from muscles.
Bohl D, Bosch A, Cardona A, Salvetti A, Heard JM.
Blood 95 (9) :2793-2798
6. Charaterization of novel lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells.
Nègre D, Mangeot PE, Duisit G, Blanchard S, Vidalain P-O, Leissner P, Winter A, Rabourdin-Combe C, Mehtali M, Moullier P, Darlix J-L, Cosset FL.
Gene Ther 7(19): 1613-162
1999
1. Functional characterization of adenoviral/retroviral chimeric vectors and their use for efficient screening of retroviral producer cell lines.
Duisit G, Salvetti A, Moullier P, Cosset F.-L.
Hum. Gene Ther. 10(2) : 189-200 (1999)
2. Baculovirus vector requires electrostatic interactions including heparan sulfate for efficient gene transfer in mammalian cells.
Duisit G, Saleun S, Douthe, S, Barsoum J, Chadeuf G, Moullier P.
J. Gene Med. 1:1-10 – (1999)
3. Efficient Gene delivery to quiescent IL2-dependent cells by murine leukemia virus-derived vectors harboring IL2 chimeric envelopes glycoproteins.
Maurice M, Mazur, S, Bullough F.J, Salvetti A, Collins M.K.L, Russell S.J, Cosset F.-L.
Blood 94(2): 401-410
4. In vivo retroviral-mediated gene transfer in the liver of dogs results in transient expression and induction of a cytotoxic immune response.
Izembart A, Aguado E, Gauthier O, Aubert D, Moullier P, Ferry N.
Hum. Gene Ther. 10(12) : 2917-2925
5. Evaluation of recombinant retrovirus and adenovirus for gene transfer to normal and pathologic intestinal tissue.
Laine F, Blouin V, Ferry N.
Gastrenterol Clin Biol. 1999
1998
1. Factors influencing adeno-associated virus production.
Salvetti A, Chadeuf G, Orève S, Favre D, Cherel Y, Champion-Arnaud P, David-Ameline J, Moullier P.
Hum. Gene Ther. 9:695-706. (1998)
2. Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector.
Bohl D, Salvetti A, Moullier P, Heard J.-M.
Blood.. 92(5):1512-1517. (1998)
3. Retrovirus vectors derived from murine leukemia viruses.
Heard J.M, Danos O, Moullier P.
The Biotherapy of cancers. Editor : S. Chouaib, Paris. Les Editions INSERM. Research in… 265-288. (1998)
4. Liver-directed gene transfer vectors.
Ferry N. and Heard JM.
Hum. Gene Ther. 9:1975-1981.
5. Mature hepatocytes actively divide and re-express gamma-glutamyl transpeptidase after D-galactosamine liver injury.
Kitten O. and Ferry N.
Liver 18. 398-404. 1998.
1997
1. Hyperosmolality suppresses but TGFb1 increases MMP9 in human peritoneal mesothelial cells.
Rougier JP, Moullier P, Piedagnel R, Ronco P.
Kidney Int. 1997, 51,120-129.
2. Principes et enjeux de la thérapie génique.
Moullier P, Ferry N, Heard J.M,
La Revue du Praticien, 1997, 47, 174-181.
3. Highly efficient retroviral-mediated gene transfer into hepatocytes in vivo.
Kitten O, Cosset FL and Ferry N (1997)
Human Gene Ther. 8: 1491-1494.
4. Gene transfer into the kidney: current status and limitations.
Moullier P, Salvetti A, Champion-Arnaud P, Ronco P.
Nephron . 1997. 77:139-151.
5. Overexpression of ecto 5’-nucleotidase promotes P-glycoprotein expression in renal epithelial cells.
Ledoux S, Leroy C, Siegfried G, Prié D, Moullier P, Friedlander G.
Kidney Int. 1997 oct;52(4):953-61
6. La thérapie génique des cancers primitifs du foie: espoirs et réalités.
Ferry N.
Bull. Cancer. 84:431-434. 1997
7. La thérapie génique.
Ferry N.
Ann. Pathol. 17 Suppl 5:29-32.
8. Thérapie génique d’un modèle de glioblastome chez le rat à l’aide d’adénovirus porteurs du gène HSVtk.
Quillien V, Heresbach N, Dufour T, Denais A, Guegan Y, Ferry N. Blouin V.
Bull. Cancer 84: 1047-1052. 1997
1996
1. Cell proliferation in chemical hepatocarcinogenesis. In vivo analysis using retroviral mediated gene transfer Lab.
Bralet MP, Calise D, Bréchot C and Ferry N. (1996)
Invest. 74: 871-881.
2. Efficient transfer of regulated genes in adipocytes and hepatoma cells by the combination of liposomes and replication defective adenovirus.
Meunier-Durmort C, Ferry N, Hainque B, Delattre J and Forest C. (1996)
Eur. J. Biochem. 237: 660-667.
3. In vivo retroviral mediated transfer of a marker gene in ornithine transcarbamylase deficient spf-ash mice.
Podevin G, Ferry N, Calise D and Révillon Y. (1996)
J. Pediatr. Surg. 31: 1516-1519.
4. In vitro and in vivo hepatoma cell specific expression of a gene transferred with an adenoviral vector.
Arbuthnot P, Bralet MP, Le Jossic C, Dedieu JF, Perricaudet M, Brechot C and Ferry N.
Human Gene Ther. 7: 1503-1514.(1996)
1995
1. Retroviral-mediated gene transfer corrects very-long-chain fatty acid metabolism in adrenoleukodystrophy fibroblasts.
Cartier N, Lopez J, Moullier P, Rocchiccioli F, Rolland MO, Jorge P, Mosser J, Mandel JL, Bougneres PF, Danos O, et al.
Proc Natl Acad Sci U S A . 1995, 92:1674-8.
2. Sustained delivery of erythropoietin in mice by genetically modified skin fibroblasts.
Naffakh N, Henri A, Villeval JL, Rouyer-Fessard P, Moullier P, Blumenfeld N, Danos O, Vainchenker W, Heard JM, Beuzard Y.
Proc Natl Acad Sci U S A . 1995, 92:3194-8.
3. Long-term delivery of a lysosomal enzyme by genetically modified fibroblasts: pre-clinical evaluation of efficacy and safety in dogs.
Moullier P, Bohl D, Cardoso J, Heard J.M. and Danos O.
Nature. Med. 1:353-357.
4. In vivo delivery of human a-L-iduronidase in mice implanted with neo-organs.
Salvetti A, Moullier P, Cornet, V, Brooks, D, Hopwood, J.J, Danos, O. and Heard, J.M.
Hum. Gene Ther. 1995, 6: 1153-1159.
5. Gene therapy of lysosomal storage disorders.
A.Salvetti , J.M. Heard, O.Danos.
British Med. Bull. 1995. 51: 106-122.
6. Functional retroviral vector for gene therapy of xeroderma pigmentosum group D patients.
M. Carreau, X. Quilliet, E. Eveno, A. Salvetti, O. Danos, J.M. Heard, M. Mezzina, A. Sarasin. .
Hum. Gene Ther. 1995. 6:1307-1315.
7. Correction of fumarylacetoacetate hydrolase deficiency (type I tyrosinemia) in cultured human fibroblasts by retroviral-mediated gene transfer.
Phaneuf D, Hadchouel M, Tanguay R.M, Bréchot C and Ferry N
Biochem. Biophys. Res. Commun. 1995 Mar 28 ; 208(3) : 957-963.
8. Hepatoma cell specific expression of a retrovirally transferred gene is achieved by alpha-fetoprotein but not insulinlike growth factor II regulatory sequences.
Arbuthnot P, Bralet M.P, Thomassin H, Danan J.L, Bréchot C and Ferry N
Hepatology. 1995 Dec ;22(6) : 1788-1796.
1994
1. Association of 1078 del T cystic fibrosis mutation with severe disease.
Moullier P, Jehanne M, Audrezet MP, Mercier B, Verlingue C, Quere I, Guillermit H, Raguenes O, Storni V, Rault G, et al.
J Med Genet . 1994, 31:159-61.
2. Adenoviral-mediated gene transfer to renal tubular cells in vivo.
Moullier P, Friedlander G, Calise D, Ronco P, Perricaudet M, Ferry N.
Kidney Int. 1994, 45:1220-5.
3. Cell lineage study in the liver using retroviral mediated gene transfer.
Bralet MP, Branchereau S, Brechot C and Ferry N (1994)
Amer. J. Pathol. 1994 May ;144(05): 896-905.
4. Factors influencing retroviral mediated gene transfer into hepatocytes in vivo.
Branchereau S, Calise D and Ferry N. (1994)
Human Gene Ther. 1994 jul ; 5(7) : 803-808.
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