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One-size-fits-all intravenous delivery of biologics may be giving way to alternate delivery routes that enable safer and more efficient drug administration. Michael Eisenstein reports.
Strategies are needed for drafting miRNA patent applications, in light of the existing patent landscape and genomic patent strategies of the past decades.
With information on the Deepwater Horizon oil spill in the Gulf of Mexico still coming in, more is being learned about the role of indigenous bacteria in cleaning the spill. Meanwhile, efforts are under way to enlist new genomic technologies to improve outcomes. Jeffrey L. Fox reports.
Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens.
The inadvertent cutting of nerves is a common adverse event during surgery. To ease visual identification of nerve fibers, Whitney et al. use phage display to develop a peptide that specifically stains peripheral nerves in living mice and in human tissue samples.
Previous work has suggested that induced pluripotent stem cells (iPSCs) are inferior to embryonic stem cells (ESCs) with respect to in vitro differentiation, raising questions about the utility of iPSCs for disease modeling. Characterization of a test set of 16 human iPSC lines shows that they perform as well as ESCs in differentiating to motor neurons.
Bantscheff et al. use chemoproteomics to measure the affinity of small molecules for megadalton protein complexes in cell extracts. Differences in the selectivity of HDAC inhibitors observed when native HDAC complexes are compared with their purified catalytic subunits suggest the limitations of using isolated recombinant proteins in certain drug screens.
TALEs (transcription activator–like effectors) contain a large number of nearly identical repeats, which makes it difficult to synthesize new variants. Feng et al. describe a facile method for assembling TALEs and show TALEs' utility for activating expression of endogenous human genes.
Synthetic biology requires methods for modular, scalable control of gene expression. Liu et al. show that unnatural amino acids can be used to regulate transcription and use the approach to create NOR and OR gates.
Ye et al. mimic a natural pathway for IgG transfer to deliver a vaccine across mucosal barriers. Intranasal immunization of mice with a fusion of a herpes simplex virus type-2 (HSV-2) antigen to an Fc fragment induces long-lasting protection after intravaginal challenge with HSV-2.
Messenger RNA has received little attention as a potential therapeutic agent. Kormann et al. show that intramuscular injection of chemically modified erythropoietin mRNA substantially increases the hematocrit in mice and demonstrate the curative potential of pulmonary mRNA delivery in a mouse model of congenital surfactant protein B deficiency.
