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Article
Open AccessAnakinra in Sanfilippo syndrome: a phase 1/2 trial
Sanfilippo syndrome is a fatal childhood neurodegenerative disorder involving neuroinflammation among multiple pathologies. We hypothesized that anakinra, a recombinant interleukin-1 receptor antagonist, could...
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Article
Open AccessCorrection to: Tet3 regulates cellular identity and DNA methylation in neural progenitor cells
The article Tet3 regulates cellular identity and DNA methylation in neural progenitor cells, written by Miguel R. Branco and C. Joana Marques, was originally published electronically on the publisher’s interne...
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Article
Open AccessTet3 regulates cellular identity and DNA methylation in neural progenitor cells
TET enzymes oxidize 5-methylcytosine (5mC) into 5-hydroxymethylcytosine (5hmC), a process thought to be intermediary in an active DNA demethylation mechanism. Notably, 5hmC is highly abundant in the brain and ...
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Protocol
Rapid Genetic Modification of Mouse Embryonic Stem Cells by Inducible Cassette Exchange Recombination
Embryonic stem cell (ESC) differentiation is a useful means by which to produce large quantities of cells in vitro representing early stages of embryonic development. A conditional gene expression system allow...
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Article
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells
Duchenne muscular dystrophy is a progressive and incurable neuromuscular disease caused by genetic and biochemical defects of the dystrophin–glycoprotein complex. Here we show the regenerative potential of myo...
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Article
BCL6 controls neurogenesis through Sirt1-dependent epigenetic repression of selective Notch targets
The authors show that the oncogene Bcl6 is necessary for proper cortical neurogenesis in vivo and from embryonic stem cells. BCL6 alters the composition of Notch-dependent transcriptional complexes at the Hes5 pr...
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Article
Generation of functional thyroid from embryonic stem cells
The primary function of the thyroid gland is to metabolize iodide by synthesizing thyroid hormones, which are critical regulators of growth, development and metabolism in almost all tissues. So far, research o...
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Article
Embryonic stem cell–based map** of developmental transcriptional programs
Presented is a study of gene regulation during development using a combination of chromatin immunoprecipitation and high-throughput sequencing (ChIP-seq) and directed differentiation of mouse embryonic stem ce...
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Article
Open AccessTranscripts that associate with the RNA binding protein, DEAD-END (DND1), in embryonic stem (ES) cells
The RNA binding protein, DEAD END (DND1), is essential for maintaining viable germ cells in vertebrates. It is also a testicular germ cell tumor susceptibility factor in mice. DND1 has been shown to interact w...
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Article
The Wnt3a/β-catenin target gene Mesogenin1 controls the segmentation clock by activating a Notch signalling program
Segmentation is an organizing principle of body plans. The segmentation clock, a molecular oscillator best illustrated by the cyclic expression of Notch signalling genes, controls the periodic cleavage of somi...
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Article
HoxA3 is an apical regulator of haemogenic endothelium
In the develo** mouse embryo, a region of the dorsal aorta endothelium generates haematopoietic cells, but how the switch to endothelium or haematopoiesis is controlled has been unclear. HoxA3 induces endoth...
