Federico Mingozzi

Evry, 91000, France

Last Updated: 09/26/2026

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Found 120 publications

Identification of alterations of immunometabolism associated with Pompe disease.

Identification of alterations of immunometabolism associated with Pompe disease.

Journal: Cellular and molecular life sciences : CMLS

Published: August 20, 2025

Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases.

Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: August 12, 2025

A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.

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

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

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

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain.

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain.

Journal: Nature communications

Published: March 26, 2024

Complement System Response to Adeno-Associated Virus Vector Gene Therapy.

Complement System Response to Adeno-Associated Virus Vector Gene Therapy.

Journal: Human gene therapy

Published: January 22, 2024

Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors.

Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors.

Journal: Human gene therapy

Published: September 06, 2023

Gene Therapy in Patients with the Crigler-Najjar Syndrome.

Gene Therapy in Patients with the Crigler-Najjar Syndrome.

Journal: The New England journal of medicine

Published: August 16, 2023

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.

Journal: Molecular metabolism

Published: July 31, 2023

Analysis of Adeno-Associated Virus Serotype 8 (AAV8)-antibody complexes using epitope mapping by molecular imprinting leads to the identification of Fab peptides that potentially evade AAV8 neutralisation.

Analysis of Adeno-Associated Virus Serotype 8 (AAV8)-antibody complexes using epitope mapping by molecular imprinting leads to the identification of Fab peptides that potentially evade AAV8 neutralisation.

Journal: Nanomedicine : nanotechnology, biology, and medicine

Published: February 01, 2023

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.

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

Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.

Successful treatment of severe MSUD in Bckdhb-/- mice with neonatal AAV gene therapy.

Journal: Journal of inherited metabolic disease

Published: November 30, 2022

Showing 1-12 of 120

Last Updated: 09/26/2026