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Archives: Publication

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

February 7, 2025

Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina

February 7, 2025

In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach

February 7, 2025

Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness

February 7, 2025

[Advances and Challenges in Microdystrophin gene therapy for Duchenne Muscular Dystrophy: progress and future directions]

February 7, 2025

[Therapeutic perspectives for lysosomal storage disorders caused by acid ceramidase deficiency]

February 7, 2025

GNT0004, Genethon’s AAV8 vector-delivered microdystrophin gene therapy of Duchenne muscular dystrophy, first data of the phase I/II part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys

January 22, 2025

An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species

December 12, 2024

Spastin regulates ER-mitochondrial contact sites and mitochondrial homeostasis

December 12, 2024

Correction of exon 2, exon 2-9 and exons 8-9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system

December 12, 2024

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