WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation

Transplant of gene-modified autologous hematopoietic progenitors cells has emerged as a new therapeutic approach for Wiskott-Aldrich syndrome (WAS), a primary immunodeficiency with microthrombocytopenia and abnormal lymphoid and myeloid functions. Despite the clinical benefits obtained in ongoing cl...

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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: Muñoz, Pilar, Tristán-Manzano, María, Sánchez-Gilabert, Almudena, Santilli, Giorgia, Galy, Anne, Thrasher, Adrian J., Martin, Francisco
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2020
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7558809/
https://ncbi.nlm.nih.gov/pubmed/33102615
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.09.006
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