AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

AAV-mediated gene therapy holds promise for the treatment of lysosomal storage diseases (LSDs), some of which are already in clinical trials. Yet, ultra-rare subtypes of LSDs, such as some glycoproteinoses, have lagged. Here, we report on a long-term safety and efficacy preclinical study conducted i...

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Autores principales: Huimin Hu, Rosario Mosca, Elida Gomero, Diantha van de Vlekkert, Yvan Campos, Leigh E. Fremuth, Scott A. Brown, Jason A. Weesner, Ida Annunziata, Alessandra d’Azzo
Formato: article
Lenguaje:EN
Publicado: Elsevier 2021
Materias:
lysosomal storage disease
galactosialidosis
protective protein cathepsin A
lysosomal multienzyme complex
AAV-mediated gene therapy
Genetics
QH426-470
Cytology
QH573-671
Acceso en línea:https://doaj.org/article/4f4d2859730e48d497c3d91716323069
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https://doaj.org/article/4f4d2859730e48d497c3d91716323069

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