Scientific Publications 2011

1. 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.

2. Biodistribution and shedding of AAV vectors.
Le Guiner C, Moullier P, Arruda VR.
Methods Mol Biol. 2011 ;807 :339-59

3. 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.

4. 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.

5. 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

6. 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

7. 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

8. 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

9. Measuring immune responses to recombinant AAV gene transfer.
Martino AT, Herzog RW, Anegon I, Adjali O.
Methods Mol Biol. 2011 ;807 :259-72

10. Methods in Molecular Biology 807, 2011 – Adeno-Associated Virus : Methods and Protocols
Richard O.Snyder, Philippe Moullier Editors.
Book

11. Evaluation of the fate of rAAV genomes following in vivo administration.
Clark KR, Penaud-Budloo M.
Methods Mol Biol. 2011 ;807 :239-258.

12. 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.

 Scientific Publications 2010

1. 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

2. 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

3. 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.

4. 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.

5. 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

6. AAV mediated gene therapy for the treatment of retinal diseases.
Rolling F.
Curr Gene Ther.2010 Oct

 Scientific Publications 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.

 Scientific Publications 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.

 Scientific Publications 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.