Scientific publications

Polyfunctional T cells and unique cytokine clusters imprint the anti rAAV2/rAAV9 vector immune response. Stephan J Holtkamp, Florian R Lagoda, Adam Lister, Pradeep Harish, Ulrike Kleymann, Theresa Pesh, Chain Fen Soon, Munir Pirmohamed, Dean Naisbitt, Mark Trautwein. Front Immunol. 2024 Nov.

Does gene therapies clinical research in rare diseases reflects the competitivity of the country: Example of France. Lutsyk K, Gicquel T, Cortial L, Forget S, Braun S, Boyer PO, Laugel V, Blin O. Therapie. 2024 Sep-Oct.

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B. Ferla R, Dell’Aquila F, Doria M, Ferraiuolo M, Noto A, Grazioli F, Ammendola V, Testa F, Melillo P, Iodice C, Risca G, Tedesco N, le Brun PR, Surace EM, Simonelli F, Galimberti S, Valsecchi MG, Marteau JB, Veron P, Colloca S, Auricchio A. Mol Ther Methods Clin Dev. 2023 Feb.

Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI. Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell’Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O’Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A.NEJM Evid. 2022 Jul.

Confirmatory detection of neutralizing antibodies to AAV gene therapy using a cell-based transduction inhibition assay. Kasprzyk T, Triffault S, Long BR, Zoog SJ, Vettermann C. Mol Ther Methods Clin Dev. 2022 Jan.

Preclinical Evaluation of a Cell-Based Gene Therapy Using the Sleeping Beauty Transposon System in Choroidal Neovascularization. Hernandez M, Recalde S, Garcia-Garcia L, Bezunartea J, Miskey C, Johnen S, Diarra S, Sebe A, Rodriguez-Madoz JR, Pouillot S, Marie C, Izsvák Z, Scherman D, Kropp M, Prosper F, Thumann G, Ivics Z, Garcia-Layana A, Fernandez-Robredo P.Mol Ther Methods Clin Dev. 2019 Nov.

Strategy to detect pre-existing immunity to AAV gene therapy. Falese L, Sandza K, Yates B, Triffault S, Gangar S, Long B, Tsuruda L, Carter B, Vettermann C, Zoog SJ, Fong S. Gene Ther. 2017 Dec.

Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. Ferla R, Alliegro M, Marteau JB, Dell’Anno M, Nusco E, Pouillot S, Galimberti S, Valsecchi MG, Zuliani V, Auricchio A. Mol Ther Methods Clin Dev. 2017 Jul.

“RCL-Pooling Assay”: A Simplified Method for the Detection of Replication-Competent Lentiviruses in Vector Batches Using Sequential Pooling. Corre G, Dessainte M, Marteau JB, Dalle B, Fenard D, Galy A. Hum Gene Ther. 2016 Feb.