Researchers say that for the first time in human beings, two of the breakthrough technologies such as stem cell technology and precision gene therapy have been united. This signifies that patients suffering from genetic disorders could be day be treated with the help of their own cells.
In existing treatment stem cells generated from a patient suffering genetic illness cannot be used to treat the disease because the cell taken from the patients would also contain the contaminated genetic code. In novel study researchers corrected stem cells mutation created from a patient suffering from a liver disease.
The research teams from the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease. The disease occurred due to alteration in a solitary duo of letters from total of six billion, which structure the genetic cipher.
Consequently, a protein called antitrypsin that protects the body from damage cannot break out from the liver where it is generated. This ailment is the most frequent genetic diseases, which affects one in every two thousand people in Europe. Liver transplant is the only solution, but this needs lifelong medicines to put off organ refusal.
For their study researchers took a skin cell from a patient and converted it to a stem cell. They used a molecular scalpel to stencil the solitary mutation and insert the precise letter for correcting the genetic error. Those stem cells were then turned into liver cells. The study was reported in Nature.
When these stem cells were inserted into mice, they were still working correctly after the duration of six weeks. According to one of the lead authors Prof David Lomas, they worked marvelously with normal secretion and function. However, the technique was absurdly tough yet the potential is enormous, but only time will tell.
More animals’ studies and human clinical trials would be required prior to any treatment because the key factor is safety, added Prof Lomas. Most gene therapy is not correcting the gene, initiating a new copy of the gene, the exciting things is that it corrects. One of the major barriers is the cost of individualized medicine, explained Prof Robin Ali, from University College London.
source : http://www.bbc.co.uk/news