A molecule that promotes brain development could serve as a possible treatment for Rett syndrome, the most common form of autism in girls, according to researchers at MIT's Picower Institute for Learning and Memory and the Whitehead Institute for Biomedical Research.
The researchers found that injecting the molecule into mice that have an equivalent of Rett syndrome helped the animals' faulty brain cells develop normally and reversed some of the disorder's symptoms.
The work, reported in the Feb. 10 online edition of the Proceedings of the National Academy of Sciences (PNAS), is expected to lead to new human clinical trials for a derivative of growth factor-1 (IGF-1), currently used to treat growth disorders and control blood glucose. The MIT study indicates that IGF-1 could potentially lessen the severity of symptoms of Rett syndrome.
"We demonstrate that a major underlying mechanism behind Rett syndrome in mice is that synapses in the brain remain immature and show persistent, abnormal plasticity into adulthood," said Daniela Tropea, a postdoctoral fellow at the Picower Institute and lead author of the study. "We also propose that a therapeutic based on this mechanism would be directly applicable to humans."
Injecting mice with a peptide fragment of IGF-1, used by the brain for neuronal and synaptic development, reverses a large number of symptoms of mice genetically engineered to display Rett syndrome-like symptoms.
"IGF-1 is critical for brain development. It activates molecules within neurons that make synapses mature," said study co-author Mriganka Sur, the Newton Professor of Neuroscience at the Picower Institute and head of the MIT Department of Brain and Cognitive Sciences. "This is a mechanism-based therapeutic for Rett syndrome. It is possible that this or similar therapeutics would apply to other forms of autism, which also have as their basis a persistent immaturity of synapses."
HELPING NERVE CELLS MATURE
Rett syndrome, an inherited neurological disorder, causes loss of speech, reduced head size, breathing and heart abnormalities and autism-like symptoms in one out of 10,000 girls.
In 85 percent of girls with Rett syndrome, the culprit is a faulty gene coding for methyl CpG-binding protein 2, (MeCP2), critical for nerve cell maturation. A deficit in MeCP2 stops neurons from growing spines, the branch-like projections needed for cell-to-cell communication.
Recent genetic studies have shown that increasing MeCP2 expression in mice led neurons to grow new spines, indicating that the disease could be reversible. Increased IGF-1 seems to make up for the lack of MeCP2.
Daily injections of the insulin-like growth factor IGF-1 extended the life spans of infant Rett syndrome mice, improved their motor function and breathing patterns and reduced irregularities in their heart rates. In addition, their brains had more nerve-cell spines.
IGF-1 affects almost every cell in the human body, especially in muscle, cartilage, bone, liver, kidney, nerves, skin and lungs. In addition to its insulin-like effects, IGF-1 also regulates cell growth and development in nerve cells.
"This is the first realistic way for a drug-like molecule injected into the bloodstream to relieve Rett syndrome symptoms," said Whitehead member Rudolf Jaenisch, whose lab participated in the research.
Elizabeth Thomson | EurekAlert!
Further reports about: > IGF-1 > MECP2 > Molecule > Neurological Disorder > Rett syndrome > Rett syndrome-like symptoms > autism in girls > autism-like symptoms > blood glucose > brain cell > breathing and heart abnormalities > faulty brain cells > loss of speech > methyl CpG-binding protein 2 > reduced head size > synaptic development
3D images of cancer cells in the body: Medical physicists from Halle present new method
16.05.2018 | Martin-Luther-Universität Halle-Wittenberg
Better equipped in the fight against lung cancer
16.05.2018 | Friedrich-Alexander-Universität Erlangen-Nürnberg
So-called quantum many-body scars allow quantum systems to stay out of equilibrium much longer, explaining experiment | Study published in Nature Physics
Recently, researchers from Harvard and MIT succeeded in trapping a record 53 atoms and individually controlling their quantum state, realizing what is called a...
The historic first detection of gravitational waves from colliding black holes far outside our galaxy opened a new window to understanding the universe. A...
A team led by Austrian experimental physicist Rainer Blatt has succeeded in characterizing the quantum entanglement of two spatially separated atoms by observing their light emission. This fundamental demonstration could lead to the development of highly sensitive optical gradiometers for the precise measurement of the gravitational field or the earth's magnetic field.
The age of quantum technology has long been heralded. Decades of research into the quantum world have led to the development of methods that make it possible...
Cardiovascular tissue engineering aims to treat heart disease with prostheses that grow and regenerate. Now, researchers from the University of Zurich, the Technical University Eindhoven and the Charité Berlin have successfully implanted regenerative heart valves, designed with the aid of computer simulations, into sheep for the first time.
Producing living tissue or organs based on human cells is one of the main research fields in regenerative medicine. Tissue engineering, which involves growing...
A team of scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg investigated optically-induced superconductivity in the alkali-doped fulleride K3C60under high external pressures. This study allowed, on one hand, to uniquely assess the nature of the transient state as a superconducting phase. In addition, it unveiled the possibility to induce superconductivity in K3C60 at temperatures far above the -170 degrees Celsius hypothesized previously, and rather all the way to room temperature. The paper by Cantaluppi et al has been published in Nature Physics.
Unlike ordinary metals, superconductors have the unique capability of transporting electrical currents without any loss. Nowadays, their technological...
02.05.2018 | Event News
13.04.2018 | Event News
12.04.2018 | Event News
18.05.2018 | Power and Electrical Engineering
18.05.2018 | Information Technology
18.05.2018 | Information Technology