An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model

Hematopoietic stem and progenitor cells (HSPCs) can establish a long‐lasting microglia‐like progeny in the central nervous system of properly myeloablated hosts. We exploited this approach to treat the severe CLN1 neurodegenerative disorder, which is the most aggressive form of neuronal ceroid lipof...

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Detalles Bibliográficos
Autores principales: Peviani, Marco, Das, Sabyasachi, Patel, Janki, Jno‐Charles, Odella, Kumar, Rajesh, Zguro, Ana, Mathews, Tyler D, Cabras, Paolo, Milazzo, Rita, Cavalca, Eleonora, Poletti, Valentina, Biffi, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10086581/
https://www.ncbi.nlm.nih.gov/pubmed/36876653
http://dx.doi.org/10.15252/emmm.202215968

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10086581/
https://www.ncbi.nlm.nih.gov/pubmed/36876653
http://dx.doi.org/10.15252/emmm.202215968

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