Gene therapy trials in seven children who suffer from Wiskott-Aldrich syndrome succeed. Six of the seven children successfully cured while the other one died because of resistance herpes.
Wiskott-Aldrich syndrome is a rare disease that afflicts 10 infants than 1 million births. The disease is easily injured but difficult to heal, susceptible to various infections including pneumonia and autoimmune.
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| The lives of six boys with a deadly genetic disease have been transformed by a pioneering treatment to correct errors in their DNA, say doctors. (Picture from: http://bbc.in/1DdOCl9) |
The cause of autoimmune disease is a mutation in the gene that carries the code for formation WAS protein. Mutations in the genes that make a protein that inhibits the formation of pieces of blood so that blood clotting is slow.
During this time, the only alternative treatment of Wiskott-Aldrich syndrome is a bone marrow transplant. However, the transplant itself is difficult because it must find a donor who really fits.
Overcoming the limitations, Salima Hacein Bey Abina of the Necker Children's Hospital try to do gene therapy. He recruited 12 children with Wiskott-Aldrich syndrome of Britain and France for trial. Of course, this is with the consent of the patient's parents.
In the therapy, Abina and her colleagues took the bone marrow and purifying it to obtain cells that affect the immune system. Once obtained, then she inserts the HIV genes that have been "tamed".
Cells that have been inserted by HIV genes were transplanted back into the spinal cord so that it can thrive. The therapeutic effect is conducted between the years of 2012 to 2014 was last seen for several months.
The results, published in the Journal of the American Medical Association on Tuesday, April 21, 2015, all children in good health after treatment and their condition is improving.
Eczema, skin problems and infection susceptibility improved in 6 patients. There was no incidence of bleeding in all patients after infection. While, autoimmune also improved in 5 patients.
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| Daniel Wheeler, who is now 15 and from Bristol, was the first British patient who were helped thanks to gene therapy. (Picture from: http://bbc.in/1DdOCl9) |
One of the patients who were helped thanks to gene therapy is Daniel Wheeler, 15 years old from Bristol. Earlier, his brother died of the same disease. Now, he could be relieved thanks to gene therapy.
Adrien Trasher of Great Ormond Street Hospital in London, as quoted by the BBC on Tuesday, saying, "I think this is very significant. This is clear evidence that an effective gene therapy approach."
However, Ian Alexander of Gene Therapy Research Unit at the Children's Medical Research Institute in Sydney, Australia, says that this is still the beginning of gene therapy. "Gene therapy is still in the infant stage and many things that were promised have not been realized," he said. *** [EKA | FROM VARIOUS SOURCES | BBC | SCIENCEDAILY]
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