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Nathalie Daniele

Genethon

SCHOLARLY PAPERS

2

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61

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0

Scholarly Papers (2)

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1.

Lipid Metabolism Is Disrupted in Mouse and Human FKRP-Deficient Muscles and Rescued by Gene Transfer

Number of pages: 57 Posted: 19 Apr 2026
Evelyne Gicquel, Marine Faivre, Manon Benabidès, Quentin Miagoux, Abbass Jaber, David Israeli, Stephany Campuzano, Margot Jarrige, Ai Vu Hong, Guillaume Corre, Laurine Buscara, Nathalie Daniele, Xavier Nissan and Isabelle Richard
Genethon, Genethon, University of Paris-Saclay, University of Paris-Saclay, Genethon, Genethon, Genethon, University of Paris-Saclay, Genethon, Genethon, Genethon, Genethon, University of Paris-Saclay and Genethon
Downloads 32 (1,411,822)

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FKRP, Limb Girdle Muscle Dystrophy, Cholesterol, AAV, Gene Transfer

2.

Shuffling of HVRs in AAV Capsid Reveals a Context-Dependent Role for HVR5 in Peripheral Tissue Detargeting While Ensuring CNS Targeting

Number of pages: 55 Posted: 12 Nov 2025
Christian Leborgne, Novella Tedesco, Bérangère Bertin, Sophie Frin, Justine Nozi, Ante Mihaljevic, Valle Montalvo-Romeral, Stephanie Astord, Anne Bigot, Nathalie Daniele, Laetitia Van Wittenberghe, Laura Cancedda, Andrea Contestabile, Maria Grazia Biferi, Piera Smeriglio, Tiziana La Bella, Giuseppe Ronzitti and Sneak Peek Administrator
Genethon, Genethon, Genethon, Genethon, Genethon, Genethon, Genethon, Sorbonne University, Sorbonne University, Genethon, Genethon, Istituto Italiano di Tecnologia, Istituto Italiano di Tecnologia, Sorbonne University, Sorbonne University, Genethon, Genethon and Independent
Downloads 29 (1,332,057)

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Adeno-associated virus, AAV gene therapy, Capsid engineering, CNS targeting, AAV variants, Vector specificity, Neurological disorders