TGF-β Pathway Inhibition Signals New Hope for Fanconi Anemia.

Tummala, Hemanth; Dokal, Inderjeet · Cell Stem Cell · 2016

basic_science · Level V

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Abstract

An improved understanding of the bone marrow failure (BMF) mechanisms in Fanconi anemia (FA) may improve current therapeutic strategies. Zhang et al. identify hyperactive TGF-β signaling as an underlying cause of BMF in FA mice and patient cells, whose inhibition promotes DNA repair and hematopoietic stem and progenitor cell survival.

Medical subject headings