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  • Diagnosing narcolepsy

    A new way to diagnose narcolepsy using T cells could help Jazz Pharmaceuticals find patients eligible to take Xyrem. In addition, GlaxoSmithKline wants to use the findings to develop influenza vaccines that lack the …

    Published on 1/23/2014
  • Silencing KRAS

    Oncogenic KRAS is notoriously difficult to target. Silenseed thinks its localized, sustained siRNA delivery technology, dubbed LODER, provides a viable strategy and also addresses issues of tissue targeting, cellular …

    Published on 1/9/2014
  • Family seeds

    In attempting to make specific microRNA inhibitors, companies have assumed that anti-miRNAs should approximate the length of the miRNA target. Now, an international team has generated in vivo proof of concept that …

    Published on 12/12/2013
  • Plan B for anti-miRNA

    How to formulate and deliver therapeutic anti-microRNAs has been a big challenge in the burgeoning anti-miRNA space. Now, a San Diego team has proof of concept for a surprisingly simple way to deliver these molecules …

    Published on 12/12/2013
  • Figure 1: Anti-miRNA delivery by B cells

    Tools: Plan B for anti-miRNA Figure 1. Anti-miRNA delivery by B cells. Almanza et al. have engineered B cells to deliver anti-microRNA to other immune cells. The findings suggest a new avenue for delivering …

    Published on 12/12/2013
  • Naive human iPS cells

    The inability to generate fully undifferentiated human iPS cells in vitro may contribute to inefficient and inconsistent differentiation of the cells. An Israeli team thinks a small molecule and cytokine cocktail …

    Published on 11/21/2013
  • Figure 1: Restoring dermal integrity

    Tools: Scleroderma models: skin in the game Figure 1. Restoring dermal integrity. A study from Gerber et al. has elucidated potential mechanisms by which mutations in the integrin-binding domain of fibrillin 1 (Fbn1)…

    Published on 11/14/2013
  • Scleroderma models: skin in the game

    North American researchers have mouse data showing that mutated fibrillin 1 recapitulates skin fibrosis and other symptoms seen in systemic scleroderma. The group also found downstream proteins whose expression was …

    Published on 11/14/2013
  • Phenotypic screening on target

    A Stanford-UCSF team has fished out a new class of NAMPT inhibitor using a phenotypic screen coupled with an shRNA-based target identification system. The method could have broad applicability in matching hits to …

    Published on 11/7/2013
  • Seeing CCR5

    A crystal structure of human CCR5 bound to the marketed HIV drug Selzentry reveals a unique binding site for the compound. The structural snapshot in combination with molecular modeling could guide future drug discovery…

    Published on 10/10/2013
  • Trapping human genes

    The Research Center for Molecular Medicine of the Austrian Academy of Sciences and Haplogen have generated a library of haploid knockout human cells that allows for the systematic investigation of gene function in vitro…

    Published on 9/26/2013
  • Figure 1: CAR-expressing T cell production to develop immunotherapeutics

    Tools: Supersizing adoptive T cell therapies Figure 1. CAR-expressing T cell production to develop immunotherapeutics. (I) Themeli et al. transduced a healthy volunteer's T cells with two retroviral vectors encoding …

    Published on 9/19/2013
  • Brain in a dish

    Austrian researchers grabbed headlines last month when they coaxed cultured human iPS cells into forming brain tissue, but in actuality the approach does not offer applications beyond studying very early brain …

    Published on 9/19/2013
  • Supersizing adoptive T cell therapies

    Studies of CAR-based T cell therapies are limited by the lack of a suitable supply of human T lymphocytes. Memorial Sloan-Kettering Cancer Center researchers have used patient-derived iPS cells for large-scale …

    Published on 9/19/2013
  • Crisper results for CRISPR

    CRISPR-based genome editing is touted for its simplicity and efficiency over other platforms, but recently published data raise concerns about its specificity. Broad Institute researchers have generated an updated …

    Published on 9/12/2013
  • Molecular biocontainment for the flu

    A U.S. team has engineered a flu virus that is transmissible among ferrets but not humans. The containment strategy provides a safeguard in case of accidental or intentional misuse of research viruses, although how well…

    Published on 9/12/2013
  • Table 1: Behavior DISCrimination

    DISCerning schizophrenia in mice Table 1. Behavior DISCrimination. Experiments are based on fixed-period sessions in which animals are placed in an enclosed chamber, and the number of times they produce a response is…

    Published on 8/29/2013
  • DISCerning schizophrenia in mice

    A team at The Johns Hopkins University has created a transgenic mouse model of prefrontal dysfunction involving disrupted networks of neurons that cause behavioral changes similar to those seen in patients with …

    Published on 8/29/2013
  • Chromosome shutdown

    A new technique to shut down chromosome 21 will reshape how Down syndrome is modeled and, in the longer term, could point to new disease targets or a strategy to remove the extra chromosome in patient cells.

    Published on 8/15/2013
  • A new wave of arrhythmia assays

    Although the race is on to select and validate new preclinical assays that the FDA will accept as replacements for current tests to predict a molecule's arrhythmia potential, the field continues to make even newer …

    Published on 8/8/2013
  • The liver's new bud

    Japanese researchers have produced vascularized human liver tissue that displays promising metabolic and regenerative capacity in mice. The group now aims to scale up the procedure to generate bigger tissue segments and…

    Published on 8/1/2013
  • Universal appeal for CARs

    MD Anderson and Sangamo are overcoming the need for autologous cells in CAR-expressing T cell-based immunotherapies. The group has used gene-modifying tools to mask donor-derived T cells from immune surveillance …

    Published on 7/25/2013
  • Cancer genome statistics lesson

    The Broad Institute has upgraded its original algorithm for cancer genome analysis to account for gene-specific differences in mutation rates. Application of the improved algorithm could help identify previously …

    Published on 7/18/2013
  • Attenuating attrition

    High drug attrition rates from poor clinical safety have spawned numerous in silico optimization algorithms for molecule design, but none has emerged as a true game changer. AstraZeneca and Roche are pooling their data …

    Published on 7/11/2013
  • Bilirubin glow

    RIKEN researchers have found a fluorescent protein in eels that potently binds bilirubin and might provide a simpler and more reliable diagnostic than existing ones. Convincing hospitals to invest in a new detection …

    Published on 6/27/2013

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