Word: adenoviruses
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...Currently, the number of iPS cells produced using retroviruses is significantly higher than that from the harmless adenoviruses. Stadtfeld, who is a post-doctoral research fellow at Harvard-affiliated Mass. General Hospital, estimated that using the more-dangerous retroviruses is 10 to 100 times more efficient than the new adenovirus method...
...control, so before testing this approach in humans, researchers need to find other modes of transport for the critical compounds. The good news is that they need the genes to churn out their proteins for a only brief period of time, so using less virulent viruses, such as the adenovirus (responsible for the common cold) or simply saturating the cell with growth factors and nutrients directly may work. "Technically, it should be possible to introduce genes or protein to reprogram cells," says Hochedlinger...
Jesse's death, for example, may have occurred because his immune system reacted violently not to the gene the scientists infused but to the virus that carried it into the cells of his liver. While deactivated, the virus--a form of the adenovirus that causes the common cold--still retained some properties capable of triggering an immune response. Indeed, what has alarmed officials at the National Institutes of Health is how common such reactions to adenovirus appear to be--and how seldom researchers have reported them to the NIH. Out of 93 gene-therapy trials using adenovirus, it was recently...
...used as packaging material for whatever gene the patient lacks. In Jesse's study, all 18 participants had the same disease: ornithine transcarbamoylase (OTC) deficiency, which slows the liver's ability to metabolize nitrogen and releases deadly ammonia into the bloodstream. So Wilson's team harnessed the adenovirus (a cause of the common cold), neutralized harmful elements and used the virus to send in normal copies of the gene that was defective in Jesse...
Most researchers in the field agree that the adenovirus and retrovirus vectors are imperfect, to say the least. In addition to having immunological side effects, both lack the carrying capacity to accommodate the larger, more complex genes that would be useful in therapy. "There are only three problems in gene therapy," says Salk's Verma, "delivery, delivery and delivery. It isn't going to be a problem to make gene therapy work--if we have an appropriate set of tools to deliver the genes...