Efficacy and Safety of a Krabbe Disease Gene Therapy

Krabbe disease is a lysosomal storage disease caused by mutations in the gene that encodes galactosylceramidase, in which galactosylsphingosine (psychosine) accumulation drives demyelination in the central and peripheral nervous systems, ultimately progressing to death in early childhood. Gene thera...

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Detalles Bibliográficos
Autores principales: Hordeaux, Juliette, Jeffrey, Brianne A., Jian, Jinlong, Choudhury, Gourav R., Michalson, Kristofer, Mitchell, Thomas W., Buza, Elizabeth L., Chichester, Jessica, Dyer, Cecilia, Bagel, Jessica, Vite, Charles H., Bradbury, Allison M., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9142772/
https://www.ncbi.nlm.nih.gov/pubmed/35333110
http://dx.doi.org/10.1089/hum.2021.245

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9142772/
https://www.ncbi.nlm.nih.gov/pubmed/35333110
http://dx.doi.org/10.1089/hum.2021.245

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