The technique, detailed in an upcoming issue of Vision Research, involves injecting the eye with a bit of genetic material called interfering RNA, which helps disable the gene.
Normally the gene is essential for healthy eyesight, but mutated versions of it are passed from generation to generation in some families and can lead to blindness.
Disabling the gene is a step toward developing a gene therapy to treat people with retinitis pigmentosa, an inherited disease that attacks the light-sensing cells in the eye. It affects about one in 60,000 people, with an estimated 1.5 million people afflicted worldwide.
"One of the causes of the disease is mutated gene expression," said Marina Gorbatyuk, Ph.D., an assistant professor of molecular genetics and microbiology in the UF College of Medicine. "We work with rhodopsin, which is the main retinal protein. Without it, or if it is mutated, people simply won’t see."
Mutated forms of the rhodopsin produce a toxic protein in the retina that kills cells that receive light. People with the disease usually notice symptoms between the ages of 10 and 30. At first they have problems seeing in dimly lit places, followed by loss of their peripheral sight. The rate of progression varies, but most patients are blind by 40.
UF Genetics Institute researchers engineered the interfering RNA into a virus, which in turn was injected below the retinas in more than a dozen normal mice. Analysis showed the technique reduced the amount of rhodopsin by about 60 percent.
With the gene drastically muzzled, scientists have begun experiments to create a therapy in which healthy versions of the gene can be introduced into the eye using an apparently harmless virus to deliver the genetic material.
"If we reduce the amount of protein formed by mutated rhodopsin, that may be sufficient to maintain vision in people who are affected by retinitis pigmentosa," Gorbatyuk said. "The second step, introducing the normal gene to the retina, will show whether we are able to restore vision in this model or not."
If both steps are perfected, scientists plan to study the treatment in a larger animal model and then possibly move to a human clinical trial.
John D. Pastor | EurekAlert!
Researchers develop eco-friendly, 4-in-1 catalyst
25.04.2017 | Brown University
Transfecting cells gently – the LZH presents a GNOME prototype at the Labvolution 2017
25.04.2017 | Laser Zentrum Hannover e.V.
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
25.04.2017 | Physics and Astronomy
25.04.2017 | Materials Sciences
25.04.2017 | Life Sciences