The latest study indicates that lentiviral gene therapy is a safe and effective approach to treat certain diseases. In this method researchers used lentiviruses base, which they have modified not only to improve the efficiency of gene transfer, but also to avoid the activation of cancer-causing genes by loading the vector with self-inactivating promoter sequences that exclusively induce the expression of the therapeutic gene. The researchers transferred a functional ARSA gene into HSCs taken from nine pre-symptomatic MLD patients. Analyses performed 2 years after treatment with the modified HSCs in one patient, and after 18 months in another two patients, revealed that 45–80 percent of the blood cells carried the functional gene. The enzyme was present at normal, healthy levels in these cells and in the cerebro-spinal fluid, where it was completely lacking before. What’s more, long after the symptoms would usually have manifested, the progression of the disease had been stopped in its tracks.
Read more: http://www.the-scientist.com/?articles.view/articleNo/36481/title/Gene-Therapy-Coming-of-Age-/
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