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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Detalles Bibliográficos
Autores principales: Hu, Huimin, Mosca, Rosario, Gomero, Elida, van de Vlekkert, Diantha, Campos, Yvan, Fremuth, Leigh E., Brown, Scott A., Weesner, Jason A., Annunziata, Ida, d’Azzo, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8640647/
https://www.ncbi.nlm.nih.gov/pubmed/34901309
http://dx.doi.org/10.1016/j.omtm.2021.10.007

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