Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal

Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged as a promising approach to achieve widespread transduction of the central nervous system (CNS) and peripheral nervous system (PNS), with direct applicability to the treatment of a wide range of neurological di...

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Detalles Bibliográficos
Autores principales: Marcó, Sara, Haurigot, Virginia, Jaén, Maria Luisa, Ribera, Albert, Sánchez, Víctor, Molas, Maria, Garcia, Miguel, León, Xavier, Roca, Carles, Sánchez, Xavier, Bertolin, Joan, Pérez, Jennifer, Elias, Gemma, Navarro, Marc, Carretero, Ana, Pumarola, Martí, Andaluz, Anna, Espada, Yvonne, Añor, Sonia, Bosch, Fatima
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/PMC8550992/
https://www.ncbi.nlm.nih.gov/pubmed/34761052
http://dx.doi.org/10.1016/j.omtm.2021.09.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8550992/
https://www.ncbi.nlm.nih.gov/pubmed/34761052
http://dx.doi.org/10.1016/j.omtm.2021.09.017

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