Editors' ChoicePharmacology

Papers of note in Science Translational Medicine

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Sci. Signal.  13 Sep 2016:
Vol. 9, Issue 445, pp. ec213
DOI: 10.1126/scisignal.aaj1832

CANCER

Stemming the regrowth of leukemia

Inhibition of BCL-2, MCL-1, and BCL-XL eradicates CML stem cells in a mouse model and primary blast crisis samples.

B. Z. Carter, P. Y. Mak, H. Mu, H. Zhou, D. H. Mak, W. Schober, J. D. Leverson, B. Zhang, R. Bhatia, X. Huang, J. Cortes, H. Kantarjian, M. Konopleva, M. Andreeff, Combined targeting of BCL-2 and BCR-ABL tyrosine kinase eradicates chronic myeloid leukemia stem cells. Sci. Transl. Med. 8, 355ra117 (2016). [Abstract]

LYSOSOMAL STORAGE DISEASES

Heat shock protein to the rescue

Increasing HSP70 expression in lysosomes using the small-molecule arimoclomol ameliorates pathology in several animal models of sphingolipidoses.

T. Kirkegaard, J. Gray, D. A. Priestman, K.-L. Wallom, J. Atkins, O. D. Olsen, A. Klein, S. Drndarski, N. H. T. Petersen, L. Ingemann, D. A. Smith, L. Morris, C. Bornæs, S. H. Jørgensen, I. Williams, A. Hinsby, C. Arenz, D. Begley, M. Jäättelä, F. M. Platt, Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses. Sci. Transl. Med. 8, 355ra118 (2016). [Abstract]

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