Scientific publications

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.