Fanny Collaud
Evry, 91000, France
Last Updated: 09/26/2026
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Found 35 publications
Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.
Journal: Acta neuropathologica communications
Published: May 06, 2026
Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiency.
Journal: Molecular therapy. Advances
Published: November 18, 2025
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.
Journal: Molecular therapy. Methods & clinical development
Published: January 09, 2025
Editorial: Gene therapy and genome editing for metabolic liver disorders.
Journal: Frontiers in molecular medicine
Published: December 11, 2024
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
Journal: Molecular therapy. Methods & clinical development
Published: April 26, 2024
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
Journal: The New England journal of medicine
Published: August 16, 2023
Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.
Journal: JCI insight
Published: May 26, 2023
Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.
Journal: Molecular therapy. Methods & clinical development
Published: May 08, 2023
Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Journal: Journal of inherited metabolic disease
Published: December 21, 2022
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Journal: Nature communications
Published: February 16, 2021
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.
Journal: Molecular therapy. Methods & clinical development
Published: September 11, 2020
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
Journal: Molecular therapy. Methods & clinical development
Published: July 09, 2020
Last Updated: 09/26/2026