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SciBX: Science-Business eXchange
As published Thursday, April 24, 2014

  • Cover Story: CRISPR in the liver

    By Amy Donner, Senior Editor

    CRISPR in the liver

    Despite the fact that the therapeutic utility of CRISPR-based approaches has yet to be demonstrated, venture dollars keep flowing into new companies developing the platform. But proof of concept may come faster than expected as new findings show that a CRIPSR-based compound can correct a mutation in adult mice with genetic liver disease.1

    Full Article
  • Reversing (heart) failure in Friedreich's ataxia

    Heart failure accounts for over half the deaths in patients with Friedreich's ataxia, but there are no therapies for the neurodegenerative disease. Now, a team of French researchers has shown that i.v. frataxin gene therapy could prevent or reverse heart failure in a mouse model of the disease.

  • Leveling cancer with IL-15

    Findings that link high IL-15 levels in tumors with increased local T cell proliferation and favorable patient outcomes position the cytokine as a robust prognostic and complement ongoing efforts to develop IL-15 as a therapeutic in colorectal cancer and other malignancies.

  • Mired in miR-25

    New evidence that miR-25 inhibition can help treat heart failure contradicts earlier results. Although the current study shows that blocking miR-25 restores SERCA2A -- and thus improves cardiac contractility -- discrepancies between the studies need to be resolved before a therapy can be developed.


  • This Week in Therapeutics

    Treating type 2 diabetes with a DPP-4 peptide vaccine; inhibiting Nav1.6 to alleviate optic neuritis; using cysteine dietary supplementation to prevent neurotoxicity associated with Huntington's disease; and more...

  • more therapeutics news
  • This Week in Techniques

    Detection of Plasmodium falciparum infection with ultrasound-based separation of red blood cells; blood-based DNA methylation signature for progressive supranuclear palsy and frontotemporal dementia; loss-of-function SMARCA4 mutations associated with small-cell carcinoma of the ovary, hypercalcemia type; and more...

  • more techniques news
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