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Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A
A hallmark of Fanconi anemia is accelerated decline in hematopoietic stem and progenitor cells (CD34 +) leading to bone marrow failure. Long-term treatment requires hematopoietic cell transplantation from an unaffected donor but is associated with potentially severe side-effects. Gene therapy to cor...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Ferrata Storti Foundation
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6278989/ https://www.ncbi.nlm.nih.gov/pubmed/29976742 http://dx.doi.org/10.3324/haematol.2018.194571 |