Publications scientifiques 2022

1. 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., Mouillier P., Dickson G., Popplewell L., Adjali O., Montanaro F. and Le Guiner C.
Gene Therapy 2022

2. 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

Publications scientifiques 2021

1. 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.

2. 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

3. 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

4. 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

5. 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.

Publications scientifiques 2020

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

2. 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.

3. 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.

4. 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

5. 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.

Publications scientifiques 2019

1. 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.

2. 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.

3.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

4. 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.

5. Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.
Marie Pouzolles, Alice Machado*, Mickael Guilbaud*, Magali Irla, Sarah Gailhac, Pierre Barennes, Daniela Cesana, Andrea Calabria, Fabrizio Benedicenti, Arnauld Serg e, Indu Raman, Quan-Zhen Li, Eugenio Montini, David Klatzmann, Oumeya Adjali, Naomi Taylor and Val erie S. Zimmermann
The journal of allergy and Clinical immunology, 2019 Sep 9. pii: S0091-6749(19)31171-6. doi: 10.1016/j.jaci.2019.08.029.

6. 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.

7. 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

8. 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

9. 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

Publications scientifiques 2018

1. 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.

2. 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.

3. Pharmacology of recombinant adeno-associated virus production.
Magalie Penaud-Budloo, Achille François, Nathalie Clément, Eduard Ayuso.
Molecular Therapy: Methods & Clinical Development (2018), doi: 10.1016/j.omtm.2018.01.002.

4. Vascular Delivery of Allogeneic MuStem Cells in Dystrophic Dogs Requires Only Short-Term Immunosuppression to Avoid Host Immunity and Generate Clinical/Tissue Benefits.
Judith Lorant, Thibaut Larcher, Nicolas Jaulin, Benoît Hedan, Aurélie Lardenois, Isabelle Leroux, Laurence Dubreil, Mireille Ledevin, Hélicia Goubin, Sophie Moullec, Jack-Yves Deschamps, Chantal Thorin, Catherine André, Oumeya Adjali, Karl Rouger
Cell Transplantation, First Published June 5, 2018, doi : 10.1177/0963689718776306

5. 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

6. 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.

7. 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

Publications scientifiques 2017

1. 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.

2. 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.

3. 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..

4. 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.

5. 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.

6. 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.

7. 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.

8. 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.

9. 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.

10. 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.

11. 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.

Publications scientifiques 2016

1. 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

2. 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

3. 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

4. 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

5. 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

6. 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

7. 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.

Mis à jour le 12 mai 2022.