Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

Neonatal AAV9-gene therapy of the lysosomal enzyme galactosylceramidase (GALC) significantly ameliorates central and peripheral neuropathology, prolongs survival, and largely normalizes motor deficits in Twitcher mice. Despite these therapeutic milestones, new observations identified the presence of...

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Detalles Bibliográficos
Autores principales: Heller, Gregory J., Marshall, Michael S., Issa, Yazan, Marshall, Jeffrey N., Nguyen, Duc, Rue, Emily, Pathmasiri, Koralege C., Domowicz, Miriam S., van Breemen, Richard B., Tai, Leon M., Cologna, Stephanie M., Crocker, Stephen J., Givogri, Maria I., Sands, Mark S., Bongarzone, Ernesto R.
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/PMC8116612/
https://www.ncbi.nlm.nih.gov/pubmed/33508430
http://dx.doi.org/10.1016/j.ymthe.2021.01.026

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8116612/
https://www.ncbi.nlm.nih.gov/pubmed/33508430
http://dx.doi.org/10.1016/j.ymthe.2021.01.026

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