Ferran Julia-Vila

Barcelona, CT, Spain

Last Updated: 09/26/2026

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Found 8 publications

Systemic delivery of AAV-GFM1 corrects COXPD1 molecular alterations in Gfm1R671C/- mice.

Systemic delivery of AAV-GFM1 corrects COXPD1 molecular alterations in Gfm1R671C/- mice.

Journal: EMBO molecular medicine

Published: October 03, 2025

Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy.

Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy.

Journal: Human gene therapy

Published: September 09, 2021

Two Novel Variants in YARS2 Gene Are Responsible for an Extended MLASA Phenotype with Pancreatic Insufficiency.

Two Novel Variants in YARS2 Gene Are Responsible for an Extended MLASA Phenotype with Pancreatic Insufficiency.

Journal: Journal of clinical medicine

Published: July 23, 2021

Therapy Prospects for Mitochondrial DNA Maintenance Disorders.

Therapy Prospects for Mitochondrial DNA Maintenance Disorders.

Journal: International journal of molecular sciences

Published: May 22, 2021

Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides.

Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides.

Journal: EBioMedicine

Published: September 17, 2020

Age-related metabolic changes limit efficacy of deoxynucleoside-based therapy in thymidine kinase 2-deficient mice.

Age-related metabolic changes limit efficacy of deoxynucleoside-based therapy in thymidine kinase 2-deficient mice.

Journal: EBioMedicine

Published: June 04, 2019

Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy.

Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy.

Journal: Human gene therapy

Published: March 23, 2019

Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy.

Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy.

Journal: Human gene therapy

Published: December 30, 2017

Showing 1-8 of 8

Last Updated: 09/26/2026