Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model.

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作者： Anna,Mateu-Bosch ^[1] ; Eulàlia,Segur-Bailach ^[1] ; Emma,Muñoz-Moreno ^[2] ; María José,Barallobre ^[1] ; Maria Lourdes,Arbonés ^[1] ; Sabrina,Gea-Sorlí ^[3] ; Frederic,Tort ^[1] ; Antonia,Ribes ^[3] ; Judit,García-Villoria ^[1] ; Cristina,Fillat ^[2]

作者单位： Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), 08036 Barcelona, Spain. ^[1] Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), 08036 Barcelona, Spain. ^[2] Instituto de Biología Molecular de Barcelona, IBMB-CSIC, Parc Científic de Barcelona, 08028 Barcelona, Spain. ^[3] Secció d'Errors Congènits del Metabolisme-IBC, Servei de Bioquímica i Genètica Molecular, Hospital Clínic, 08028 Barcelona, Spain. ^[4] Facultat de Medicina i Ciències de la Salut. Universitat de Barcelona, 08036 Barcelona, Spain. ^[5]

关键词 AAV adeno-associated virus gene replacement glutaric aciduria metabolic disease viral vectors

DOI 10.1016/j.omtm.2024.101276

PMID 38983872

发布时间 2024-07-11

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Molecular therapy. Methods & clinical development

2024年32卷3期

101276页

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Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

相关期刊

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