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There has been much focus on DNA mutations in tumorigenesis and tumor progression, but now a new study highlights a role for post-transcriptional changes in RNA in cancer. The authors show increased RNA editing of antizyme inhibitor 1 (AZIN1) in tumor tissues from patients with hepatocellular carcinoma and characterize a mechanism through which the expression of edited AZIN1 protein leads to increased cell proliferation (pages 209–216).
BH3 mimetics are a class of anticancer agents that hold the promise to trigger the central apoptotic machinery to set cancer cells on the road to ruin. Now, a new agent that selectively targets BCL-2, ABT-199, has been developed, with exciting preclinical and clinical results (pages 202–208).
It's up to stakeholders at every stage of therapeutic development—industry and academic researchers, policymakers, patient foundations and even patients themselves—to embrace the power of collaboration. Only then will we enable translational research and push much-needed treatments to the clinic faster.
The potential threat of parasite resistance to current antimalarials begs further research into antimalarial drug discovery to control disease progression. In addition, even when effective drugs are used, severe malaria symptoms still pose an important risk for death and cerebral residual disease in children. Further understanding of the pathophysiology of malaria and the biology of the parasite will open doors to new antimalarial treatments.
Malaria's death toll has been reduced as a result of global efforts over the last decade. Yet the rise of drug resistance and the plateauing of funding are still obstacles to eradicating the disease and reducing malaria burden. This review brings up the goals and challenges faced by researchers and the public health workforce and a way forward to effectively control and eliminate malaria.
There is renewed enthusiasm in developing an HIV vaccine and in understanding the requirements to elicit broadly neutralizing HIV-specific antibodies. In May 2012, a workshop convened researchers to discuss the interplay of CD4+ T cell and antibody responses to help identify key questions and areas of research that can inform future vaccine development. This Perspective summarizes the discussion of three main topics on the role of CD4+ T cells in HIV vaccine design.
Wnt signaling is a major regulator during development. Genetic mutations affecting main regulators of this pathway have also emphasized the relevance of Wnt signaling in bone homeostasis after birth and diseases involving bone loss and fragility, such as osteoporosis. New therapies targeting Wnt signaling to increase bone formation are now under development.
Usher syndrome is a congenital form of deafness and vestibular dysfunction characterized by mutations in the USH1C gene encoding harmonin. Michelle L. Hastings and her colleagues show in a mouse model of Usher syndrome that early treatment with antisense oligonucleotides corrects defective pre-mRNA splicing of mutant USH1C, increasing full-length harmonin expression and restoring low-frequency hearing and vestibular function.
Mutations in the cytosolic 5′-nucleotidase II gene accelerate the inactivation of chemotherapeutic nucleoside analogs in acute lymphoblastic leukemia (ALL)-promoting lymphoblasts. Increased nucleotide metabolism may therefore constitute an important resistance mechanism in chemotherapy-resilient ALL.
John Chute and his colleagues show that the cytokine EGF protects mouse bone marrow hematopoietic stem cells from radiation injury. EGF signaling in these cells inhibited cell death through repression of the proapoptotic protein PUMA. EGF administration rescued mice from death after total-body irradiation, suggesting a new therapeutic strategy for radioprotection.
Human epididymis protein 4 (HE4), a putative serine protease inhibitor, is upregulated in human and mouse fibrotic kidneys. Inhibiting HE4 inhibited fibrosis in three different mouse models of renal disease, suggesting that HE4 is a new therapeutic target.
Michalina Gora and her colleagues have developed a tethered capsule endoscope in the form of a swallowable pill that does not require sedation and is the size of a one-cent coin. Once swallowed, the device was well tolerated and used to capture three-dimensional microstructural images of the digestive tract, particularly the esophagus, using optical frequency domain imaging. Feasibility was demonstrated in patients with Barrett’s esophagus, including high-grade dysplasia.