A delivery system used in the laboratory to carry foreign DNA into a cell. Modified viruses are commonly used as vectors in gene therapy.
Scientists have successfully used gene therapy to alleviate the symptoms of the blood disease haemophilia B in six human volunteers, raising hope for a potential cure. The study has been hailed as a landmark trial for gene therapy...
US scientists have induced long-lasting HIV protection in mice from a single injection. Their study, published in the journal Nature, uses gene therapy to stimulate production of antibodies against the virus...
Scientists at the University of California San Diego (UCSD), US, have developed a technique to transfer modified genes known to cause disease in humans into human embryonic stem cell (ES cell) lines. The modified ES cells behave like diseased cells and can be used to study human genetic diseases in the laboratory. The new technique provides an alternative approach to mouse 'knock out' models of disease....
Gene therapy has been used to treat two young boys with a devastating and fatal brain disease called adrenoleukodystrophy (ALD). Two years after treatment, both boys showed signs that the disease had stopped progressing and that there were no serious side effects from the gene therapy. These results, published in the journal Science, show huge promise, both for the future treatment of ALD and for the revival of investigations into the use of gene therapy to treat a wide variety ...
Donor lungs damaged before transplantation can be repaired using a gene therapy technique developed by a team of scientists in Canada. This may increase the supply of functional lungs for transplant patients, by using organs that are currently discarded....
US doctors have used gene therapy to restore significant vision in 12 patients who were previously blinded by a disease called Leber's congenital amaurosis. The study, conducted by researchers at the Children`s Hospital of Philadelphia and the University of Pennsylvania School of Medicine, was published online in the Lancet on 24 October....
Japanese researchers announced last week that they have advanced their understanding and ability to safely 'reprogramme' adult stem cells to resemble embryonic stem (ES) cells without inducing tumours or harmful genetic abnormalities. The Japanese team of researchers, lead by Dr Shinya Yamanaka at Kyoto University, reprogrammed liver...
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