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...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2021
Soggetti:
Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8116612/
https://ncbi.nlm.nih.gov/pubmed/33508430
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2021.01.026
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi