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U.K. researchers develop gene therapy from skin cells that makes Alpha-1 liver cells work properly in mice
Medpage Today
Researchers are reporting they can correct the mutation that can cause an inherited liver disease, at least in the lab and in animals.
The report, online in Nature, is “proof of principle” that stem cells can be used as therapy for Alpha-1 Antitrypsin Deficiency and perhaps for other diseases caused by a single mutation, according to Ludovic Vallier, PhD, of Cambridge University, one of the co-authors of the study.
David Lomas, MD, PhD
The research team included David Lomas, MD, PhD, a member of the Medical and Scientific Advisory Committee of the Alpha-1 Foundation.
Vallier and other authors told reporters at a London press briefing that more research needs to be done before the novel approach they used can be used to treat patients with the condition – about 100,000 in the U.S. Alpha-1 can cause lung disease in adults and liver disease in both adults and children.
But, they noted, their approach is a step forward – they were able to correct both copies of the defective gene without introducing potentially dangerous genetic changes elsewhere in the genome.
“We needed to correct both copies of the bad gene,” said co-author Allan Bradley, PhD, of the Wellcome Trust Sanger Institute in Cambridge, England. And it had to be done, he added, in such a way that “the genome of the cells was not damaged in any way.”
The researchers began with skin cells from patients afflicted with Alpha-1 Antitrypsin Deficiency, which affects one in 2,000 people of Northern European descent and is the most common inherited metabolic disease of the liver.
