Scientific publications

Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency.
Mével M, Pichard V, Bouzelha M, Alvarez-Dorta D, Lalys PA, Provost N, Allais M, Mendes A, Landagaray E, Ducloyer JB, Toublanc E, Galy A, Brument N, Lefevre GM, Gouin SG, Isiegas C, Le Meur G, Cronin T, Le Guiner C, Weber M, Moullier P, Ayuso E, Deniaud D, Adjali O.
Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101187. doi: 10.1016/j.omtm.2024.101187. eCollection 2024 Mar 14.

Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina.
Leray A, Lalys PA, Varin J, Bouzelha M, Bourdon A, Alvarez-Dorta D, Pavageau K, Depienne S, Marchand M, Mellet A, Demilly J, Ducloyer JB, Girard T, Fraysse B, Ledevin M, Guilbaud M, Gouin SG, Ayuso E, Adjali O, Larcher T, Cronin T, Le Guiner C, Deniaud D, Mével M.
Biomed Pharmacother. 2024 Feb;171:116148. doi: 10.1016/j.biopha.2024.116148. Epub 2024 Jan 16.

Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors.
Xicluna R, Avenel A, Vandamme C, Devaux M, Jaulin N, Couzinié C, Le Duff J, Charrier A, Guilbaud M, Adjali O, Gernoux G. Hum Gene Ther. 2024 May;35(9-10):355-364. doi: 10.1089/hum.2023.225. Epub 2024 Apr 19. PMID: 38581431

Dystrophin deficiency impairs cell junction formation during embryonic myogenesis.
Mozin E, Massouridès E, Mournetas V, Lièvre C, Bourdon A, Jackson DL, Packer JS, Seong J, Trapnell C, Le Guiner C, Adjali O, Pinset C, Mack DL, Dupont JB. bioRxiv [Preprint]. 2024 Apr 8:2023.12.05.569919. doi: 10.1101/2023.12.05.569919. PMID: 38106055

Dystrophin deficiency impairs cell junction formation during embryonic myogenesis.
Mozin E, Massouridès E, Mournetas V, Lièvre C, Bourdon A, Jackson DL, Packer JS, Trapnell C, Guiner CL, Adjali O, Pinset C, Mack DL, Dupont JB.
bioRxiv [Preprint]. 2023 Dec 7:2023.12.05.569919. doi: 10.1101/2023.12.05.569919.

Management of safety issues arising following AAV gene therapy.
261st ENMC International Workshop 17th-19th June 2022, Hoofddorp, The Netherlands.
Servais L, Horton R, Saade D, Bonnemann C, Muntoni F; 261st ENMC workshop study group.
Neuromuscul Disord. 2023 Nov;33(11):884-896. doi: 10.1016/j.nmd.2023.09.008. Epub 2023 Oct 1.

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
Le Guiner C, Xiao X, Larcher T, Lafoux A, Huchet C, Toumaniantz G, Adjali O, Anegon I, Remy S, Grieger J, Li J, Farrokhi V, Neubert H, Owens J, McIntyre M, Moullier P, Samulski RJ.
Mol Ther Methods Clin Dev. 2023 May 18;30:30-47. doi: 10.1016/j.omtm.2023.05.017. eCollection 2023 Sep 14.

Transcriptomic Analysis Reveals the Inability of Recombinant AAV8 to Activate Human Monocyte-Derived Dendritic Cells.
Masri S, Carré L, Jaulin N, Vandamme C, Couzinié C, Guy-Duché A, Dupont JB, Pereira A, Charpentier E, David L, Gernoux G, Guilbaud M, Adjali O.
Int J Mol Sci. 2023 Jun 21;24(13):10447. doi: 10.3390/ijms241310447.

Analytical ultracentrifugation sedimentation velocity for the characterization of recombinant adeno-associated virus vectors sub-populations.
Saleun S, Mas C, Le Roy A, Penaud-Budloo M, Adjali O, Blouin V, Ebel C.
Eur Biophys J. 2023 Jul;52(4-5):367-377. doi: 10.1007/s00249-023-01650-3. Epub 2023 Apr 28.

Click-electrochemistry for the rapid labeling of virus, bacteria and cell surfaces.
Depienne S, Bouzelha M, Courtois E, Pavageau K, Lalys PA, Marchand M, Alvarez-Dorta D, Nedellec S, Marín-Fernández L, Grandjean C, Boujtita M, Deniaud D, Mével M, Gouin SG.
Nat Commun. 2023 Aug 23;14(1):5122. doi: 10.1038/s41467-023-40534-0.

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMD rat model
Bourdon A., François V., Zhang L., Lafoux A., Fraysse B., Toumaniantz G., Larcher T., Girard T., Ledevin M., Lebreton C., Hivonnair A., Creismeas A., Allais M., Marie B., Guguin J., Blouin V., Rémy S., Anegon I., Huchet C., Malerba A., Kao B., Le Héron A., Moullier P., Dickson G., Popplewell L., Adjali O., Montanaro F. and Le Guiner C.
Gene Ther (2022). https://doi.org/10.1038/s41434-022-00317-6
 

The Diversity of Parvovirus Telomeres
Marianne Laugel, Emilie Lecomte, Eduard Ayuso, Oumeya Adjali, Mathieu Mével and Magalie Penaud-BudlooBlouin
Recent Advances in Canine Medicine - DOI: 10.5772/intechopen.102684 - April 27th, 2022

Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity
Tran NT, Lecomte E, Saleun S, Namkung S, Robin C, Weber K, Devine E, Blouin V, Adjali O, Ayuso E, Gao G, Penaud-Budloo M, Tai PWL.
Hum Gene Ther. 2022 Apr;33(7-8):371-388. doi: 10.1089/hum.2022.050

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, Kajaste-Rudnitski A, Montini E, Penaud-Budloo M, Naldini L.
Cell Stem Cell. 2022 Oct 6;29(10):1428-1444.e9. doi: 10.1016/j.stem.2022.09.001.

Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution.
Ducloyer JB, Pichard V, Mevel M, Galy A, Lefevre GM, Brument N, Alvarez-Dorta D, Deniaud D, Mendes-Madeira A, Libeau L, Le Guiner C, Cronin T, Adjali O, Weber M, Le Meur G.
Mol Ther Methods Clin Dev. 2023 Feb 15;28:387-393. doi: 10.1016/j.omtm.2023.02.006. eCollection 2023 Mar 9.

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice.
Calabria A, Cipriani C, Spinozzi G, Rudilosso L, Esposito S, Benedicenti F, Albertini A, Pouzolles M, Luoni M, Giannelli S, Broccoli V, Guilbaud M, Adjali O, Taylor N, Zimmermann VS, Montini E, Cesana D.
Blood. 2023 May 11;141(19):2316-2329. doi: 10.1182/blood.2022017378.

AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation
Gwladys Gernoux , Mickaël Guilbaud , Marie Devaux , Malo Journou , Virginie Pichard , Nicolas Jaulin , Adrien Léger , Johanne Le Duff , Jack-Yves Deschamps , Caroline Le Guiner , Philippe Moullier , Yan Cherel , Oumeya Adjali
Mol Ther Methods Clin Dev. 2021 Feb 6;20:660-674. doi: 10.1016/j.omtm.2021.02.003. eCollection 2021 Mar 12.

Homologous Recombination Offers Advantages over Transposition-Based Systems to Generate Recombinant Baculovirus for Adeno-Associated Viral Vector Production
Jacob A, Brun L, Jiménez Gil P, Ménard L, Bouzelha M, Broucque F, Roblin A, Vandenberghe LH, Adjali O, Robin C, François A, Blouin V, Penaud-Budloo M, Ayuso E.
Biotechnol J. 2021 Jan;16(1):e2000014. doi: 10.1002/biot.202000014. Epub 2020 Nov 16.PMID: 33067902

The SSV-Seq 2.0 PCR-Free Method Improves the Sequencing of Adeno-Associated Viral Vector Genomes Containing GC-Rich Regions and Homopolymers
Lecomte E, Saleun S, Bolteau M, Guy-Duché A, Adjali O, Blouin V, Penaud-Budloo M, Ayuso E.
Biotechnol J. 2021 Jan;16(1):e2000016. doi: 10.1002/biot.202000016. Epub 2020 Nov 8.PMID: 33064875

Genetic diseases in the omics era
Jean-Baptiste Dupont,
Mol Ther. 2021 Aug 4;29(8):2389-2390. doi: 10.1016/j.ymthe.2021.07.007. Epub 2021 Jul 23

TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD
Anna Creisméas, Claire Gazaille, Audrey Bourdon, Marc-Antoine Lallemand, Virginie François, Marine Allais, Mireille Ledevin, Thibaut Larcher, Gilles Toumaniantz, Aude Lafoux, Corinne Huchet, Ignacio Anegon, Oumeya Adjali, Caroline Le Guiner, Bodvaël Fraysse
J Transl Med. 2021 Dec 20;19(1):519. doi: 10.1186/s12967-021-03191-9.

Luminol anchors improve the electrochemical-tyrosine-click labelling of proteins.
Depienne S, Alvarez-Dorta D, Croyal M, Temgoua RCT, Charlier C, Deniaud D, Mével M, Boujtita M, Gouin SG.
Chem Sci. 2021 Nov 10;12(46):15374-15381. doi: 10.1039/d1sc04809k. eCollection 2021 Dec 1.

Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8 + T Cells in Human Donors Allows the Detection of a T EMRA Subpopulation.
Vandamme C, Xicluna R, Hesnard L, Devaux M, Jaulin N, Guilbaud M, Le Duff J, Couzinié C, Moullier P, Saulquin X, Adjali O.
Front. Immunol., 21 January 2020. doi: 10.3389/fimmu.2019.03110

Tyrosine Conjugation Methods for Protein Labelling
Dimitri Alvarez Dorta, David Deniaud, Mathieu Mével, Sébastien G Gouin
Chemistry. 2020 Jun 15. doi: 10.1002/chem.202001992. Online ahead of print.

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Karim Bey, Johan Deniaud, Laurence Dubreil, Béatrice Joussemet, Joseph Cristini, Carine Ciron, Juliette Hordeaux, Morwenn Le Boulc’h, Kevin Marche, Maud Maquigneau, Michaël Guilbaud, Rosalie Moreau, Thibaut Larcher, Jack-Yves Deschamps, Marion Fusellier, Véronique Blouin, Caroline Sevin, Nathalie Cartier, Oumeya Adjali, Patrick Aubourg, Philippe Moullier, and Marie-Anne Colle
Mol Ther Methods Clin Dev. 2020 Apr 11;17:771-784. doi: 10.1016/j.omtm.2020.04.001. eCollection 2020 Jun 12.

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmdmdx rat model.
Caudal D, François V, Lafoux A , Ledevin M,  Anegon I,  Le Guiner C , Larcher T, Huchet C.
PLoS One 2020 Mar 11;15(3):e0230083. doi : 10.1371/journal.pone.0230083

The Contractile Phenotype of Skeletal Muscle in TRPV1 Knockout Mice is Gender-Specific and Exercise-Dependent.
Lafoux A, Lotteau S, Huchet C, Ducreux S.
Life (Basel). 2020 Oct 6;10(10):E233. doi: 10.3390/life10100233.

2019

Single-Stranded DNA Virus Sequencing (SSV-Seq) for Characterization of Residual DNA and AAV Vector Genomes.
Lecomte E, Leger A, Penaud-Budloo M, Ayuso E.
Methods Mol Biol. 2019;1950:85-106. doi: 10.1007/978-1-4939-9139-6_5.

5 years of successful inducible transgene expression following locoregional AAV 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 P1, Le Guiner C, Adjali O
Hum Gene Ther. 2019 Feb 27. doi: 10.1089/hum.2018.234.

Stability of the adeno-associated virus 8 reference standard material.
Penaud-Budloo M, Broucque F, Harrouet K, Bouzelha M, Saleun S, Douthe S, D'Costa S, Ogram S, Adjali O, Blouin V, Lock M, Snyder RO, Ayuso E.
Gene Ther. Mar 29. doi: 10.1038/s41434-019-0072-9. 2019

Intrinsic Differential Scanning Fluorimetry for Fast and Easy Identification of Adeno-Associated Virus Serotypes.
Rieser R, Penaud-Budloo M, Bouzelha M, Rossi A, Menzen T, Biel M, Büning H, Ayuso E, Winter G, Michalakis S.
J Pharm Sci. 2019 Oct 19. doi:10.1016/j.xphs.2019.10.031.

Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.
Pouzolles M, Machado A*, Guilbaud M*, Irla M, Gailhac S, Barennes P, Cesana D, Calabria A, Benedicenti F, Serge A, Raman I, Li QZ, Montini E, Klatzmann D, Adjali O, Taylor N and Zimmermann VS
The journal of allergy and Clinical immunology, 2019 Sep 9. pii: S0091-6749(19)31171-6. doi: 10.1016/j.jaci.2019.08.029.

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries.
Schmit PF, Pacouret S, Zinn E, Telford E, Nicolaou F, Broucque F, Andres-Mateos E, Xiao R, Penaud-Budloo M, Bouzelha M, Jaulin N, Adjali O, Ayuso E, Vandenberghe LH
Mol Ther Methods Clin Dev. 2019 Nov 26;17:107-121. doi : 10.1016/j.omtm.2019.11.014. eCollection 2020 Jun 12.

Chemical modification of adeno-associated virus capsid to improve gene delivery
Mével M, Bouzelha M, Leray A, Pacouret S, Guilbaud M, Penaud-Budloo M, Alvarez-Dorta D, Dubreuil L, Gouin SG, Combal JP, Hommel M, Gonzalez-Aseguinolaza G, Blouin V, Moullier P, Adjali A, Deniaud D, Ayuso E.
Chemical Science, 9 December 2019, doi : 10.1039/C9SC04189C

O-GlcNAc stimulation: A new metabolic approach to treat spetic shock.
Ferron M, Cadiet J, Persello A, Prat V, Denis M, Erraud A, Aillerie V, Mével M, Bogot E, Chatham JC, Gauthier C, Rozec B, Lauzier B.
Nature scientific report, 10 December 2019, doi : 10.1038/s41598-019-55381-7

Immunophenotype of a Rat Model of Duchenne's Disease and Demonstration of Improved Muscle Strength After Anti-CD45RC Antibody Treatment.
Ouisse LH, Remy S, Lafoux A, Larcher T, Tesson L, Chenouard V, Guillonneau C, Brusselle L, Vimond N, Rouger K, Péréon Y, Chenouard A, Gras-Le Guen C, Braudeau C, Josien R, Huchet C, Anegon I.
Front Immunol. 2019 Sep 9;10:2131. doi: 10.3389/fimmu.2019.02131. eCollection 2019

2018

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.

Pharmacology of recombinant adeno-associated virus production.
Penaud-Budloo M, François A, Clément N, Ayuso E.
Molecular Therapy: Methods & Clinical Development (2018), doi: 10.1016/j.omtm.2018.01.002.

Vascular Delivery of Allogeneic MuStem Cells in Dystrophic Dogs Requires Only Short-Term Immunosuppression to Avoid Host Immunity and Generate Clinical/Tissue Benefits.
Lorant J, Larcher T, Jaulin N, Hedan B, Lardenois A, Leroux I, Dubreil L, Ledevin M, Goubin H, Moullec S, JY, Thorin C, André C, Adjali O, Rouger K
Cell Transplantation, First Published June 5, 2018, doi : 10.1177/0963689718776306

The Assembly-Activating Protein Promotes Stability and Interactions between AAV’s Viral Proteins to Nucleate Capsid Assembly.
Anna Maurer, Simon Pacouret, Ana Karla Cepeda Diaz, Jessica Blake, Eva Andres-Mateos, Luk Vandenberghe
Cell Reports , Elsevier Inc, 23 (6), pp.1817 - 1830, doi : 10.1016/j.celrep.2018.04.026

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.

Electrochemically Promoted Tyrosine-Click-Chemistry for Protein Labelling
Alvarez-Dorta D, Thobie-Gautier C, Croyal M, Bouzelha M, Mével M, Deniaud D, Boujtita M, Gouin SG
J Am Chem Soc. 2018 Nov 28. doi: 10.1021/jacs.8b09372

2017

An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21-/- mouse model
Perdomini M, Dos Santos C, Goumeaux C, Blouin V, Bougnères P.
Gene Ther. 2017 Feb 10.

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 C2, Tournaire B, Cogné B, Léger A, Blouin V, Lindenbaum P, Moullier P, Ayuso E. Hum Gene Ther Methods. 2017 May 2. doi: 10.1089/hgtb.2016.185.

Outer Plexiform Layer Structures Are Not Altered Following AAV-Mediated Gene Transferin Healthy Rat Retina.
Giers C, Klein D, Mendes-Madeira A, Isiegas C, Lorenz B, Haverkamp S, Stieger K.
Frontiers in Neurology. 2017-Feb-23..

Relevance of Assembly-Activating Protein for Adeno-associated Virus Vector Production and Capsid Protein Stability in Mammalian and Insect Cells.
Grosse S, Penaud-Budloo M, Herrmann AK1, Börner K1, Fakhiri J, Laketa V, Krämer C, Wiedtke E, Gunkel M, Ménard L, Ayuso E, Grimm D.
J Virol. 2017 Sep 27;91(20). pii: e01198-17. doi: 10.1128/JVI.01198-17. Print 2017 Oct 15.

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.

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial.
Vandamme C, Adjali O, Mingozzi F.
Hum Gene Ther. 2017 Nov;28(11):1061-1074. doi: 10.1089/hum.2017.150.

Gait characterization in golden retriever muscular dystrophy dogs using linear discriminant analysis.
Fraysse B, Barthélémy I, Qannari EM, Rouger K, Thorin C, Blot S, Le Guiner C, Chérel Y, Hogrel JY.
BMC Musculoskelet Disord. 2017 Apr 12;18(1):153. doi: 10.1186/s12891-017-1494-4.

Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders.
Bey K, Ciron C, Dubreil L, Deniaud J, Ledevin M, Cristini J, Blouin V, Aubourg P, Colle MA.
Gene Ther. 2017 May 24.

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.

Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.
Hordeaux J, Dubreil L, Robveille C, Deniaud J, Pascal Q, Dequéant B, Pailloux J, Lagalice L, Ledevin M, Babarit C, Costiou P, Jamme F, Fusellier M, Mallem Y, Ciron C, Huchet C, Caillaud C, Colle MA.
Acta Neuropathol Commun. 2017 Sep 6;5(1):66. doi: 10.1186/s40478-017-0464-2.

Skeletal muscle relaxant effect of a standardized extract of Valeriana officinalis L. after acute administration in mice.
Caudal D, Guinobert I, Lafoux A, Bardot V, Cotte C, Ripoche I, Chalard P, Huchet C.
J Tradit Complement Med. 2017 Oct 12;8(2):335-340

2016

Manufacturing of recombinant AAV vectors: new technologies are welcome
Ayuso E.
Mol Ther Methods & Clinical Development (2016) 3, 15049; doi:10.1038/mtm.2015.49

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 Feb 9. doi: 10.1038/mt.2016.37. [Epub ahead of print] PMID: 26857842

Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR.
PD’Costa S, Blouin V, Broucque F, Penaud-Budloo M, A François, IC. Perez, Le Bec C, Moullier P, Snyder RO and Ayuso E.
Mol Ther Methods Clin Dev. 2016 Mar

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

Seipin deficiency alters brown adipose tissue thermogenesis and insulin sensitivity in a non-cell autonomous mode.
Dollet L, Magré J, Joubert M, Le May C, Ayer A, Arnaud L, Pecqueur C, Blouin V, Cariou B, Prieur X.
Sci Rep. 2016 Oct

A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts.
McClements ME, Charbel Issa P, Blouin V, MacLaren RE.
J Genet Syndr Gene Ther. 2016 Nov 14

Soluble Milk Protein Supplementation with Moderate Physical Activity Improves Locomotion Function in Aging Rats.
Lafoux A, Baudry C, Bonhomme C, Le Ruyet P, Huchet C.
PLoS One. 2016 Dec 14;11(12):e0167707. doi: 10.1371/journal.pone.0167707. eCollection 2016.

2015

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

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

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.

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.

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

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.

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

    Before 2010