Scientific publications 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.

Scientific publications 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

Scientific publications 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.

 Scientific publications 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