Sentences with phrase «targeted human gene therapy»
The first successful targeted human gene therapy was reported in 2000.
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Before moving on to human trials, they will need to study all instances of «off - target» effects: Years before Crispr, the viruses employed to deliver DNA in gene therapy trials occasionally damaged the whole system, causing cancer.
In humans, variants of the IL - 15R - alpha gene have been found in world - class endurance competitors, suggesting a target for gene therapies aimed at boosting the ability to exercise longer.
This is a great example of a potential gene therapy target that still needs a fair amount of work to validate the thesis and the initial data, but having a large number of existing human carriers is a good sign on the safety front.