Long-term gene therapy resulted in improved cardiac function and reversed deterioration of the heart in rats with heart failure, according to a recent study.

A new anti-cancer compound that works by blocking a part of the cell's machinery that is crucial for cell division has shown promising results in a phase I clinical trial in patients who have failed to respond to other treatments.

Boys with Duchenne Muscular Dystrophy will usually lose the ability to walk by their teens and typically die before the age of 30. For years, scientists have studied the use of gene therapy as a possible way to correct the muscle deterioration, but hurdles such as the need to treat all muscles in the body, including both skeletal muscle and heart muscle, have challenged researchers looking for an effective therapy until now.

Researchers have identified a simple gene-based blood test that more accurately and quickly measures cystic fibrosis patients' response to therapy than current tests. The test, a measure of inflammatory gene expression, could improve patient care and help clear a backlog of promising medications now hung up in clinical trials.

Scientists have launched a phase 1 clinical trial to test whether a pain-relieving gene can be sent to a key point in the nervous system and block pain sensation. The technique promises a more effective, targeted way to treat persistent pain than present painkillers.

A new clinical trial suggests that long-term use of candesartan, a drug currently used to treat hypertension, may significantly reduce the symptoms of genetic heart disease.

Heart failure patients have four times the risk of fractures and 6.3 times the risk of hip fracture as other heart patients. Heart failure patients should be screened and treated for osteoporosis if necessary, researchers said.

Gene marker indicates doubling of survival time in advanced colorectal cancer treated with cetuximab. A study shows value of circulating tumor cells in patients on targeted therapy.

Researchers have initiated a Phase I clinical trial in London, UK to test a prime-boost combination of two HIV vaccine candidates. The news follows promising results for one of the two vaccine candidates to be tested, the MVA-based TBC-M4, which in a recent phase I trial generated modest immune responses in all volunteers who received the highest dose.

No significant adverse effects were reported during a safety trial testing gene therapy on three patients with a type of hereditary blindness called Leber congenital amaurosis type 2. In addition, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.