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Modeling Human Bone Marrow Failure Syndromes Using Pluripotent Stem Cells and Genome Engineering

The combination of epigenetic reprogramming with advanced genome editing technologies opened a new avenue to study disease mechanisms, particularly of disorders with depleted target tissue. Bone marrow failure syndromes (BMFS) typically present with a marked reduction of peripheral blood cells due t...

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Publicat a:Mol Ther
Autors principals: Jung, Moonjung, Dunbar, Cynthia E, Winkler, Thomas
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2015
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4700116/
https://ncbi.nlm.nih.gov/pubmed/26435409
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.180
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