P
Philippe Veron
Researcher at French Institute of Health and Medical Research
Publications - 16
Citations - 3300
Philippe Veron is an academic researcher from French Institute of Health and Medical Research. The author has contributed to research in topics: Genetic enhancement & Biology. The author has an hindex of 9, co-authored 12 publications receiving 2882 citations. Previous affiliations of Philippe Veron include Université Paris-Saclay & Curie Institute.
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Journal ArticleDOI
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
David L. Mack,Karine Poulard,Melissa A. Goddard,Virginie Latournerie,Jessica M. Snyder,Robert W. Grange,Matthew Elverman,Jérôme Denard,Philippe Veron,Laurine Buscara,Christine Le Bec,Jean-Yves Hogrel,Annie G. Brezovec,Hui Meng,Lin Yang,Fujun Liu,Michael O'callaghan,Nikhil Gopal,Valerie E. Kelly,Barbara K. Smith,Jennifer L. Strande,Fulvio Mavilio,Alan H. Beggs,Federico Mingozzi,Michael W. Lawlor,Ana Buj-Bello,Martin K. Childers +26 more
TL;DR: Results indicate that systemic gene therapy was well tolerated, prolonged lifespan, and corrected the skeletal musculature throughout the body in a dose-dependent manner, defining an efficacious dose in this large-animal model of the disease.
Journal ArticleDOI
Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Bérangère Bertin,Philippe Veron,Christian Leborgne,Jack-Yves Deschamps,Sophie Moullec,Yves Fromes,Fanny Collaud,Sylvie Boutin,Virginie Latournerie,Laetitia van Wittenberghe,Benoit Delache,Roger Le Grand,Nathalie Dereuddre-Bosquet,Olivier Benveniste,Philippe Moullier,Carole Masurier,Otto Wilhelm Merten,Federico Mingozzi +17 more
TL;DR: An AAV-specific plasmapheresis column was developed which was shown to efficiently and selectively deplete anti-AAV antibodies without depleting the total immunoglobulin pool from plasma, providing proof-of-concept of a method for the A AV-specific depletion of neutralizing antibodies in the setting of in vivo gene transfer.
Journal ArticleDOI
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.
Fanny Collaud,Giulia Bortolussi,Laurence Guianvarc’h,Sem J. Aronson,Thierry Bordet,Philippe Veron,Severine Charles,Patrice Vidal,Marcelo Simon Sola,Stephanie Rundwasser,Delphine G. Dufour,Florence Lacoste,Cyril Luc,Laetitia van Wittenberghe,Samia Martin,Christine Le Bec,Piter J. Bosma,Andrés F. Muro,Giuseppe Ronzitti,Matthias Hebben,Federico Mingozzi +20 more
TL;DR: An (ss)AAV8 vector carrying the human UDP-glucuronosyltransferase family 1-member A1 (hUGT1A1) transgene under the control of a liver-specific promoter is developed, supporting the clinical translation of this gene therapy approach for the treatment of Crigler-Najjar (CN) syndrome.
Journal ArticleDOI
Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A.
Brian Long,Philippe Veron,Philippe Veron,Klaudia Kuranda,Romain Hardet,Nina Mitchell,Gregory M. Hayes,Wing Yen Wong,Kelly Lau,Mingjin Li,M. Benjamin Hock,Stephen J. Zoog,Christian Vettermann,Federico Mingozzi,Federico Mingozzi,Becky Schweighardt +15 more
TL;DR: Up to 3 years of immunogenicity data following administration of valoctocogene roxaparvovec (BMN 270), an AAV5-mediated gene therapy encoding human B domain-deleted FVIII (hFVIII-SQ) in a phase 1/2 clinical study of adult males with severe hemophilia A.