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Papers of note in Science Translational Medicine 9 (413)

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Sci. Signal.  31 Oct 2017:
Vol. 10, Issue 503, eaar3223
DOI: 10.1126/scisignal.aar3223

This week’s articles describe new therapeutic targets for pulmonary arterial hypertension, acute myeloid leukemia, and Friedreich’s ataxia.

LUNG DISEASE

Progress for PAH

Michelakis et al. found that restoring the activity of the mitochondrial enzyme pyruvate dehydrogenase with inhibitors of the kinase PDK alleviated pulmonary arterial hypertension in lung explants and patients but not in patients with reduced-function variants of SIRT3 and UCP2.

CANCER

Capturing the codependencies

Saito et al. show that concurrent inhibition of the antiapoptotic factor BCL-2 and the mutant kinase FLT3 treats drug-resistant acute myeloid leukemia.

NEURODEGENERATIVE DISEASE

Cell therapy for Friedreich’s ataxia

Rocca et al. report that transplanting healthy hematopoietic stem cells into a mouse model of Friedreich’s ataxia restores microglia-to-neuron and macrophage-to-myocyte transfer of the mitochondrial proteins frataxin and Cox8 to ameliorate the symptoms of the neurodegenerative disease.

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