The controversial study proves there is hope for treating serious diseases from the womb, giving hope to unborn babies through gene therapy.
Using gene therapy scientists managed to treat hemophilia and "bubbly boy" disease, although some subjects' bodies activate the immune system and fight the treatment. Another concern is the risk of developing other diseases at the price of treating a certain disease.
Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in particular. Gene therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
So British researchers came with the solution to treat unborn babies, whose immune system is not yet capable of fighting the new genes.
Dr Simon Waddington of University College London explained: "There are several advantages. For example, in cystic fibrosis, lung damage is actually occurring before birth. So, if you can get your gene therapy in before then, you might be able to stop the disease from happening. If you are going to treat adults it is often too late to reverse some of the damage. We are not modifying the children's children, only treating that patient."
"The technologies are there to deliver and inject genes into babies if we find it is effective enough and safe enough."
Charles Coutelle, Imperial College: "The safety issue is something that will take time...and needs looking at very carefully."
Department of Health representative: "No human clinical trials of in utero gene therapy have ever taken place in the UK, nor is this considered to be feasible in the next few years."
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