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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs) engineered with integrating vectors is a promising treatment for monogenic diseases, but lack of centralized databases is hampering an overall outcomes assessment. Here we aim to provide a comprehensive assessment of the...

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Detalles Bibliográficos
Autores principales: Tucci, Francesca, Galimberti, Stefania, Naldini, Luigi, Valsecchi, Maria Grazia, Aiuti, Alessandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8921234/
https://www.ncbi.nlm.nih.gov/pubmed/35288539
http://dx.doi.org/10.1038/s41467-022-28762-2