High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing

Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological benefit in rare diseases, which is achieved through the engraftment of genetically modified microglia-like cells (MLCs) in the brain. Still, the engraftment dynamics and the nature of engineered MLCs, as well as t...

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Detalles Bibliográficos
Autores principales: Plasschaert, Robert N., DeAndrade, Mark P., Hull, Fritz, Nguyen, Quoc, Peterson, Tara, Yan, Aimin, Loperfido, Mariana, Baricordi, Cristina, Barbarossa, Luigi, Yoon, John K., Dogan, Yildirim, Unnisa, Zeenath, Schindler, Jeffrey W., van Til, Niek P., Biasco, Luca, Mason, Chris
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9552809/
https://www.ncbi.nlm.nih.gov/pubmed/35614857
http://dx.doi.org/10.1016/j.ymthe.2022.05.022

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