Some genetically engineered viruses were used to correct harmful mutations in DNA
Italian doctors announced a cure for a disease that takes away the ability of children to walk and talk. The study, announced in the journal Science, used a pioneering technique of gene therapy to correct errors in DNA.
Three patients in the study are already going to school.
A second study, published at the same time showed that a similar therapy can reverse a severe genetic disease that affects the immune system.
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Gene therapy researchers say the result of the studies is "really exciting."
Both diseases are caused by errors in the genetic code of the patient - responsible for the growth and functioning of the body. Babies born with metachromatic leukodystrophy appear healthy, but its development starts to regress between one and two years old, as part of your brain is destroyed.
The Wiskott-Aldrich results in an impaired immune system. She makes patients more susceptible to infections, cancers, and immune system itself can also attack other parts of the body.
Infected cells
The technique developed by the research team of the San Raffaele Scientific Institute in Milan, Italy, used a genetically modified virus to correct harmful mutations in genes of patients.
Stem cells are taken from the patient and the virus is used to "infect" cells with small DNA fragments that contain the correct instructions. These are then placed back into the patient.
Three children from families with a history of metachromatic leukodystrophy were selected for treatment, but before your brain function began to degenerate.
Alessandra Biffi, one of the doctors involved in the study, told the BBC: "The result was very positive, all are well, and living a normal life at an age when his brothers were unable to speak.'s A result that makes us very happy" .
Biffi said that all treatments have side effects, and patients had to be followed for longer. But evidence suggests so far that the treatment is safe.
'New era'
Gene therapy is a field that promised much more than it has delivered and has been hampered by several security concerns. The first gene therapy in Europe was only approved in 2012.
Biffi said that lessons have been learned from previous failures: "Gene therapy can be improved. We may be living in a new era where we can do more than we did in the past."
In another study, published simultaneously in the journal Science, as infections and eczema symptoms had decreased in three patients treated.
Source: http://www.uol.com.br
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