Researchers used tiny, magnetic spheres to deliver a gene editing toolbox into stem cells prepared from a patient’s skin sample and correct a genetic mutation known to cause Rett syndrome. When the edited stem cells were given cues to turn into neurons (nerve cells) in the lab, they looked…
Stem cells engineered to produce a constant level of a downstream target of the MECP2 protein, called brain-derived neurotrophic factor (BDNF), increased the development of new neurons and prevented body weight loss in a mouse model of Rett syndrome. The study with that finding, “Brain-Derived Neurotrophic Factor Secreting…
Genes that are normally silenced in the X-chromosome transition from an “off’ to an “on” state at different speeds, an event that is dependent on the action of certain proteins and enzymes, according to a recent study. These findings may one day help design a targeted therapeutic strategy for…
Loss of MECP2, the defective protein in most people with Rett syndrome, induces premature growth arrest of bone marrow stem cells — known as senescence — and limits their energy production, according to a mouse study. These biological alterations may underlie the development of the disease, the researchers…
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