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

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

Abstract 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 cer...

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Bibliographic Details
Main Authors: Marco Peviani, Sabyasachi Das, Janki Patel, Odella Jno‐Charles, Rajesh Kumar, Ana Zguro, Tyler D Mathews, Paolo Cabras, Rita Milazzo, Eleonora Cavalca, Valentina Poletti, Alessandra Biffi
Format: Article
Language:English
Published: Springer Nature 2023-03-01
Series:EMBO Molecular Medicine
Subjects:
CLN1
hematopoietic stem cell gene therapy
lysosomal storage disorder
microglia
Online Access:https://doi.org/10.15252/emmm.202215968

Internet

https://doi.org/10.15252/emmm.202215968

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