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Vision-saving gene therapy; longer living fruit flies; cell transplants in mice

This is a recurring column on early-stage research in animals or other laboratory models that has not entered the clinic yet but could have implications for future research and development of human medicines.

Rats and macaques and ... a Yorkshire pig, oh my???

SNL Image
Researchers with Johns Hopkins Medicine developed a new technique to conduct gene therapy in the eyes of rats, monkeys and pigs.
Source: Pixabay

Researchers with Johns Hopkins Medicine are searching for a way to save the sight of humans by injecting harmless, genetically engineered viruses into the eyes of rats, Rhesus macaques and a Yorkshire pig.

The research team developed a gene therapy technique that successfully prevented vision loss in all three animals and could prove to be a successful treatment for humans with eye conditions like wet age-related macular degeneration, or AMD, and inherited retinal disease, according to the study, published in The Journal of Clinical Investigation.

The "wet" form of AMD causes blurred vision and sight loss from excess fluid in the eye due to the overproduction of the cell signal vascular endothelial growth factor, or VEGF.

The researchers blocked the production of VEGF, and ensured that a virus carrying anti-VEGF genes could make it to the retina. By injecting the virus into the space between the white of the eye and its vascular layer, researchers were able to reach the retina with the therapeutic genes.

This technique is safer for patients and less invasive than a more complex method that requires a surgical procedure to inject gene-carrying viruses under the retina, which is used to treat the eye disorder Leber congenital amaurosis, according to Johns Hopkins.

Researchers first tested if the gene therapies could reach the retina by using the technique to deliver the adeno-associated virus to 10 rats, then used the virus to deliver the anti-VEGF gene.

Forty rats induced to have a human form of macular degeneration were injected with the anti-VEGF gene using the new technique, and another 40 were injected using an older technique. The researchers found that the new technique successfully delivered the anti-VEGF gene, protected the vision of the rats and was just as effective as the previous technique.

To test the new technique on larger animals with an eye size similar to humans, the team conducted the experiment on Rhesus macaques and a Yorkshire pig, getting similar results.

Peter Campochiaro, a researcher on the study, said in a Sept. 30 statement that if the new delivery technique proved successful in humans, treatment could one day be as simple as "walking into a clinic."

Extending lifespan of fruit flies

Scientists with the University College London and the Max Planck Institute for Biology of Ageing may have found a fountain of youth of sorts in a triple-drug combination treatment that extended the lifespan of fruit flies.

The study found that fruit flies lived 48% longer with a combined treatment of psychiatric medication lithium, chemotherapy trametinib and immunosupressant rapamycin compared to no treatment, according to the study published Sept. 30 in Proceedings of the National Academy of Sciences. Single drug treatment extended lifespans by an average of 11% for each drug, and two-drug treatments extended lifespan by about 30%.

Linda Partridge, principal investigator on the study, said treatment may be an effective way to slow down the aging process in humans and keep patients healthy in their old age.

"We are not trying to cheat death, but help people be healthy and disease-free in their final years," Partridge said in an Oct. 1 press release.

SNL ImageResearchers developed a method to transplant brain cells in mice without anti-rejection drugs.
Source: AP Photo

Stem cell transplants in mice

A research team with Johns Hopkins Medicine successfully transplanted protective brain cells in mice without needing anti-rejection drugs to keep the cells alive, according to a study published in the neurology journal Brain.

Scientists focused on transplanting protective brain cells that create myelin sheaths, the layer around neurons that helps them transmit electrical signals. The method may eventually help treat patients with genetic diseases that stop the normal formation of myelin.

The transplantation of tissues and cells requires anti-rejection drugs to prevent the immune system from attacking the new tissues. The drugs can lead to health risks because they depress a patient's immune system.

Scientists in the study first injected mouse brains with glial cells that produce the myelin sheath. The team then put the cells in normal mice, mice genetically engineered to not create the cells and mice bred to be unable to launch an immune response. Certain mice were then injected with antibodies to stop immune systems from attacking the new cells.

Mice that received the antibodies maintained glial cells for 203 days after they were transplanted, and the cells successfully created myelin sheaths, according to the study.

Piotr Walczak, researcher and associate professor of radiology and radiological science at the Johns Hopkins University School of Medicine, said the results were preliminary and seen only in a localized portion of the mouse brain.