SYDNEY, Nov. 16 (Xinhua) -- Australian researchers on Friday rolled out a major gene therapy treatment program aimed at saving children suffering from life-threatening genetic liver disease.
The program involves the development of new viral vectors, which transport a healthy copy of a gene into a patient's cells to either replace or edit faulty ones, the Children's Medical Research Institute said in a statement.
"We're trying to get to a point where instead of a liver transplant in a very young infant, we can genetically repair the liver without major surgery." said program leader Professor Ian Alexander.
"This technology could translate into saving the lives of infants with life-threatening conditions. It's about getting cures into the clinic as soon as we can."
The program is in line with a broad-ranging New South Wales state health and medical initiative to develop gene transfer vectors, according to the institute.
"At the moment, we can routinely produce vectors in the lab, but to move to much larger clinical scales it is very costly and involves long, overseas waiting times for clinical trials," said institute researcher Dr Leszek Lisowski.
"That means the therapies are not going to be available to most of the public any time soon. We aim to change that; to reduce costs and local waiting times for trials so these cures are more widely and more quickly available.
"While the initial focus is on liver the technology has the potential to expand to other disease targets, involving the brain, the heart, kidneys, bone marrow and pancreas, for example."
