Stem cells used to grow liver cells
Scientists have developed a method for growing functional liver cells from a patient’s own skin cells by combining embryonic stem cells and gene therapy techniques.
A team of UK researchers has used skin cells of patients suffering from a genetic liver disease - alpha1-antitrypsin (AAT) deficiency - and successfully transformed them into healthy and functional liver cells.
AAT deficiency is a common inherited disorder in which liver cells produce abnormal forms of alpha1-antitrypsin, crucial for lung function. Long term, the disease affects the liver and the lungs; the patient has no option other than eventual transplants of the damaged organs.
In this study, scientists took stem cells made from the skin cells of a group of AAT deficiency patients and used "molecular scissors" to repair the gene mutation that causes the disease.
The genetically repaired stem cells were then used for developing fully functioning liver cells, which worked normally in the lab and after being transplanted into mice.
The new method may one day replace costly liver transplants which requires a suitable organ donor and even then may fail due to rejection of the transplanted organ by recipient’ s body.
In addition, those who receive a transplant must take immune suppression medication for the rest of their lives. The new therapy may provide a liver of a patient’s own cells - no rejection risk.
”We have developed new systems to target genes and integrated all the components to correct, efficiently, defects in patient cells,” said co-author Professor Allan Bradley, Director Emeritus of the Wellcome Trust Sanger Institute. ”Our systems leave behind no trace of the genetic manipulation, save for the gene correction.”
”As there is currently no cure for this disease other than liver transplantation, and given the increasing strains being placed on the national liver transplant program as a result of the sharp increase in the frequency of liver disease, alternative therapies for genetic and other liver diseases are urgently being sought.”
However, the new stem cell plus gene therapy technique has to undergo several years of trials before being used in humans. If approved, it opens a new vision in providing patient-specific, personalized treatment for genetic disorders and chronic disease, wrote the scientists in the journal Nature.
”What we are thinking about now is how we can take this through to humans, accepting that safety is paramount. The beauty of our approach is that we can make the genetic correction and we can do it cleanly,” said co-author David Lomas, deputy director of the Cambridge Institute for Medical Research.
Source: presstv.ir
