Researchers analysed thousands of laboratory-made plasmids and discovered that nearly half of them had defects, raising questions of experimental reproducibility.
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For many, many years we’ve been hearing about gene therapy – the chance that we can get into people’s DNA and fix it to resolve problems and fix disease. In a recent piece in Science, Stuart Orkin and Philip Reilly discuss what finally achieving success might mean:
Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited neurodegenerative disease. For decades, gene therapy has tantalized us with such futuristic scenarios. However, these goals are now coming into focus, and it is the time to consider some of the consequences of success.
As they report, gene therapy has been forty-four years in the making. But gene therapy, which has cost billions of dollars in research and development, is different from the traditional pharmaceutical market. For one thing, most diseases which are the focus of gene therapy research are relatively...