Scientific Publications 2015

1. 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, Mendes-Madeira A, Rolling F, Cherel Y, Moullier P, Colle M.
Gene Ther. 2015

2. Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxydative 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, O Snyder R, Moullier P, Léger A.
Molecular Therapy – Methods and clinical Development (2015) 2, 15010

3. Advanced characterization of DNA molécules in rAAV vector préparations by single-stranded virus next-generation sequencing. Mol Ther Nucleic Acids.
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.
2015 Oct 27;4:e260. doi: 10.1038/mtna.2015.32.

4. 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.

5. 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, 26 : 1-13, DOI 10.1089/hum.2014.070

6. Identification of miRNAs Involved in Reprogramming Acinar Cells into Insulin Producing Cells.
Teichenne J, Morró M, Casellas A, Jimenez V, Tellez N, Leger A, Bosch F, Ayuso E.
PLoS One. 2015 Dec 21;10(12):e0145116. doi: 10.1371/journal.pone.0145116.

Scientific Publications 2014

1. 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.

2. 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)

3. 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

4. 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.

5. 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.

6. 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

7. 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.

8. 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.

9. 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.

10. 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]

11. 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.

12. 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.

13. 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.

14. 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.

 Scientific Publications 2013

1. 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).

2. 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

3. 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

 Scientific Publications 2012

1. 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

2. 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.

3. 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

4. Recombinant adeno-associated viral vector reference standards.
Moullier P, Snyder RO.
Methods Enzymol.2012 ;507 :297-311

5. 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

6. 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

7. 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.

8. 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

9. 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