News of Note—Gene therapy to restore sight; umbilical cord cells to repair hearts; studying a Parkinson’s drug in cancer

Scientists at Oxford have developed a gene therapy technique to treat retinitis pigmentosa, a leading cause of blindness.

Gene therapy for reversing blindness

Researchers at Oxford University used gene therapy to reverse retinitis pigmentosa in mouse models of the disease, which is a common cause of blindness in young people. They delivered a gene that makes the light-sensing protein melanopsin to the remaining retinal cells of mice that had already lost their sight. Over the next year, the animals maintained a high level of vision as the cells that received the therapeutic gene responded to light and sent visual signals to the brain. The researchers are planning a clinical trial to test the technique in humans. (Release)

Repairing broken hearts with umbilical cord stem cells

In a clinical trial in Chile, patients with heart failure who were given infusions of umbilical cord-derived stem cells showed an improvement in their hearts’ pumping ability, as well as daily functioning and quality of life. Stem cells extracted from bone marrow had previously been tested in heart failure, but cord-derived cells from donated human placenta are less likely to cause immune reactions, the researchers believe. They published their findings in the American Heart Association journal Circulation Research. (Release)

Repurposing a Parkinson’s drug for pancreatic cancer

Carbidopa, a drug used to treat Parkinson’s disease, inhibited cancer cell growth in human pancreatic cancer cells and mouse models of the disease. The research, performed by scientists at Texas Tech University Health Sciences Center, suggests that the drug acts by activating the aryl hydrocarbon receptor (AhR) protein, which may have anticancer effects. The researchers hope to partner with oncologists to design a clinical trial in people. (Release)


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