Mucopolysaccharidosis (MPS) type VII (also known as Sly syndrome) is a lysosomal storage disorder (LSD) characterized by a deficiency of the lysomal enzyme beta-glucuronidase. MPS VII leads to bone and joint abnormalities, enlargement of the visceral organs, cardiovascular disease and neurologic impairment. Using a MPS VII adult animal model, researchers in the Center for Gene Therapy at Columbus Children’s Research Institute (CCRI) on the campus of Columbus Children’s Hospital have demonstrated a gene therapy strategy whereby in vivo administration into the liver of a recombinant adeno-associated virus (rAAV) type 2 vector is capable of reversing the progression of storage in the liver, spleen, kidney, heart, lung and, importantly, the brain. This is the first time researchers have demonstrated that the peripheral in vivo administration of a therapeutic gene leads to improvement in the brain of an adult MPS VII animal model. The findings were published in the September issue of Molecular Therapy.
Previous work has shown that similar therapies are effective in treating the brain if administered soon after birth, but until now, it was believed that the blood-brain barrier would prevent this occurrence during adulthood.
“It’s not clearly understood why in newborns, the blood-brain barrier is ‘open,’ and as a person matures, very few substances can cross through this barrier,” said Thomas J. Sferra, M.D., gastroenterologist and associate professor at The Ohio State University College of Medicine and Public Health. “In our study, we were able to show an improvement in the manifestations of this disease within the brain. Now that we have proven that this type of therapy can overcome the blood-brain barrier, we can begin to study how this occurred. In the long-term, we have the potential to apply this type of therapy to a majority of the LSDs and even other disorders.”
Bare bones: Making bones transparent
27.04.2017 | California Institute of Technology
Link Discovered between Immune System, Brain Structure and Memory
26.04.2017 | Universität Basel
More and more automobile companies are focusing on body parts made of carbon fiber reinforced plastics (CFRP). However, manufacturing and repair costs must be further reduced in order to make CFRP more economical in use. Together with the Volkswagen AG and five other partners in the project HolQueSt 3D, the Laser Zentrum Hannover e.V. (LZH) has developed laser processes for the automatic trimming, drilling and repair of three-dimensional components.
Automated manufacturing processes are the basis for ultimately establishing the series production of CFRP components. In the project HolQueSt 3D, the LZH has...
Reflecting the structure of composites found in nature and the ancient world, researchers at the University of Illinois at Urbana-Champaign have synthesized thin carbon nanotube (CNT) textiles that exhibit both high electrical conductivity and a level of toughness that is about fifty times higher than copper films, currently used in electronics.
"The structural robustness of thin metal films has significant importance for the reliable operation of smart skin and flexible electronics including...
The nearby, giant radio galaxy M87 hosts a supermassive black hole (BH) and is well-known for its bright jet dominating the spectrum over ten orders of magnitude in frequency. Due to its proximity, jet prominence, and the large black hole mass, M87 is the best laboratory for investigating the formation, acceleration, and collimation of relativistic jets. A research team led by Silke Britzen from the Max Planck Institute for Radio Astronomy in Bonn, Germany, has found strong indication for turbulent processes connecting the accretion disk and the jet of that galaxy providing insights into the longstanding problem of the origin of astrophysical jets.
Supermassive black holes form some of the most enigmatic phenomena in astrophysics. Their enormous energy output is supposed to be generated by the...
The probability to find a certain number of photons inside a laser pulse usually corresponds to a classical distribution of independent events, the so-called...
Microprocessors based on atomically thin materials hold the promise of the evolution of traditional processors as well as new applications in the field of flexible electronics. Now, a TU Wien research team led by Thomas Müller has made a breakthrough in this field as part of an ongoing research project.
Two-dimensional materials, or 2D materials for short, are extremely versatile, although – or often more precisely because – they are made up of just one or a...
20.04.2017 | Event News
18.04.2017 | Event News
03.04.2017 | Event News
27.04.2017 | Life Sciences
27.04.2017 | Physics and Astronomy
27.04.2017 | Earth Sciences