Two successful gene therapy trials block inherited diseases in humans (important news)
Genetic traits, like a bulbous nose or balding, give some people reasons to moan about what they inherited from their parents. But more serious genetic flaws can cause debilitating disease. Now, Italian researchers have come up with a way of treating one such inherited disease and reversing another using a promising new method of gene therapy.
The idea behind gene therapy is to replace a faulty gene with a shiny new version that works properly. Modified versions of viruses, which have been sculpted by millions of years of evolution, perfectly penetrate human cells. They act as courriers delivering DNA payloads to defective cells and ensure they are stably inherited.
This deceptively simple idea, though, has been challenging to achieve in practice. The first commercial gene therapy product, Glybera, only received regulatory approval in 2012.
Part of the reason for this is the difficulty of successfully clearing three hurdles at once: delivering replacement genetic information to the exact cells that need help, getting this information safely translated in high enough volumes to overcome the defects, and stopping the immune system from reacting to “normal” genes when it has grown used to only seeing mangled ones.
Now, a team led by Alessandra Biffi at the San Raffaele Scientific Institute in Milan, Italy, reports in Science that they have developed a new approach that navigates each of these hurdles to treat three children with metachromatic leukodystrophy (MLD), a devastating inherited disease that affects around 1 in 40,000 people.
http://arstechnica.com/science/2013/07/two-successful-gene-therapy-trials-block-inherited-diseases-in-humans/
Read the entire article, worth it.
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