Gloria Aseguinolaza-Gonzalez

Pamplona, NC 31008, Spain

Last Updated: 09/26/2026

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Found 115 publications

Optimized lentiviral vectors a solution for paediatric metabolic liver diseases.

Optimized lentiviral vectors a solution for paediatric metabolic liver diseases.

Journal: Journal of hepatology
Published: June 17, 2026

Correction: VTX-PID as a novel recombinant immunoglobulin G-degrading enzyme (IdeS) for efficient AAV-based gene therapy in participants with neutralizing antibodies: results of the phase I first-in-human NAVIgATE study.

Correction: VTX-PID as a novel recombinant immunoglobulin G-degrading enzyme (IdeS) for efficient AAV-based gene therapy in participants with neutralizing antibodies: results of the phase I first-in-human NAVIgATE study.

Journal: Frontiers in immunology
Published: June 03, 2026

Liver-directed gene therapy results in amelioration of progressive familial intrahepatic cholestasis type 2 in mice.

Liver-directed gene therapy results in amelioration of progressive familial intrahepatic cholestasis type 2 in mice.

Journal: Hepatology communications
Published: March 20, 2026

VTX-PID as a novel recombinant immunoglobulin G-degrading enzyme (IdeS) for efficient AAV-based gene therapy in participants with neutralizing antibodies: results of the phase I first-in-human NAVIgATE study.

VTX-PID as a novel recombinant immunoglobulin G-degrading enzyme (IdeS) for efficient AAV-based gene therapy in participants with neutralizing antibodies: results of the phase I first-in-human NAVIgATE study.

Journal: Frontiers in immunology
Published: March 06, 2026

The future of gene therapy for inborn errors of metabolism: insights into liver-targeted innovations.

The future of gene therapy for inborn errors of metabolism: insights into liver-targeted innovations.

Journal: Expert opinion on biological therapy
Published: January 16, 2026

Locus coeruleus α-synuclein overexpression induces prodromal Parkinsonian features in mice.

Locus coeruleus α-synuclein overexpression induces prodromal Parkinsonian features in mice.

Journal: NPJ Parkinson's disease
Published: December 17, 2025

Rapid generation of prion disease models using AAV-delivered PrP variants in knockout mice.

Rapid generation of prion disease models using AAV-delivered PrP variants in knockout mice.

Journal: Brain pathology (Zurich, Switzerland)
Published: December 01, 2025

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

Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expression.

Orthogonal characterization of rAAV reveals vector attributes that drive ITR repair, self-complementary genome formation, and transgene expression.

Journal: Molecular therapy. Nucleic acids
Published: October 01, 2025

From Radiocopper to Cold Copper: Mechanistic Modeling and Simulation to Define Clinical Response Criteria and Biomarkers for VTX-801 in Wilson Disease.

From Radiocopper to Cold Copper: Mechanistic Modeling and Simulation to Define Clinical Response Criteria and Biomarkers for VTX-801 in Wilson Disease.

Journal: CPT: pharmacometrics & systems pharmacology
Published: August 08, 2025

DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery.

DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery.

Journal: Molecular therapy. Advances
Published: August 04, 2025

Loss of PLA2G4E compromises synaptic structure and cognitive outcomes in mice.

Loss of PLA2G4E compromises synaptic structure and cognitive outcomes in mice.

Journal: Life science alliance
Published: March 25, 2025
Showing 1-12 of 115

Last Updated: 09/26/2026