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Showing 1–4 of 4 results
Advanced filters: Author: Dwayne L. Barber Clear advanced filters

  • Treatments for Fabry disease, an inherited lysosomal disorder caused by the deficiency of the enzyme alpha-galactosidase A, are not fully efficacious. Here the authors report a single-arm phase I trial of gene therapy with autologous, lentivirus-transduced, hematopoietic cells that express alpha-galactosidase A to demonstrate that this approach is safe in five patients with Fabry disease.

    • Aneal Khan
    • Dwayne L. Barber
    • Jeffrey A. Medin
    ResearchOpen Access
    Nature Communications
    Volume: 12, P: 1-9

  • Individuals with Chuvash polycythemia have an increased red blood cell count and are prone to developing blood clots. Although mutations affecting the VHL protein are known to be causative, the underlying molecular mechanisms have been unclear. Ryan C Russell et al. now show that VHL targets the key signaling molecule JAK2 for degradation by forming a complex with the SOCS1 protein. The authors also show that JAK2 inhibition has beneficial effects in a mouse model of this disease, pointing to a new therapeutic strategy.

    • Ryan C Russell
    • Roxana I Sufan
    • Michael Ohh
    Research
    Nature Medicine
    Volume: 17, P: 845-853

  • The term myelodysplastic syndromes covers various diseases that are caused by ineffective haematopoiesis in one or more lineages of the bone marrow. How do these diseases arise, and what are the best methods for treating these patients?

    • Seth J. Corey
    • Mark D. Minden
    • Aaron D. Schimmer
    Reviews
    Nature Reviews Cancer
    Volume: 7, P: 118-129

  • Mutations in isocitrate dehydrogenases IDH1 and IDH2 are common in human gliomas and acute myeloid leukaemias; here, mice that carry the IDH1(R132H) mutation are described, in a new model that should help in investigating the links between mutant IDH1 and leukaemia.

    • Masato Sasaki
    • Christiane B. Knobbe
    • Tak W. Mak
    Research
    Nature
    Volume: 488, P: 656-659