Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Gaucher disease type 1 (GD1) is an inherited lysosomal disorder with multisystemic effects in patients. Hallmark symptoms include hepatosplenomegaly, cytopenias, and bone disease with varying degrees of severity. Mutations in a single gene, glucosidase beta acid 1 (GBA1), are the underlying cause fo...

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Detalles Bibliográficos
Autores principales: Dahl, Maria, Smith, Emma M.K., Warsi, Sarah, Rothe, Michael, Ferraz, Maria J., Aerts, Johannes M.F.G., Golipour, Azadeh, Harper, Claudia, Pfeifer, Richard, Pizzurro, Daniella, Schambach, Axel, Mason, Chris, Karlsson, Stefan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7806948/
https://www.ncbi.nlm.nih.gov/pubmed/33511245
http://dx.doi.org/10.1016/j.omtm.2020.11.018

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