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Genome editing, a next step in genetic therapy, corrects hemophilia in animals
Date:6/26/2011

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The researchers injected mice with the gene therapy vector, which was designed to travel to the liverwhere clotting factors are produced. The mice that received the ZFN/gene combination then produced enough clotting factor to reduce blood clotting times to nearly normal levels. Control mice receiving vectors lacking the ZFNs or the F9 minigene had no significant improvements in circulating factor or in clotting times.

The improvements persisted over the eight months of the study, and showed no toxic effects on growth, weight gain or liver function, clues that the treatment was well-tolerated.

"We established a proof of concept that we can perform genome editing in vivo, to produce stable and clinically meaningful results," said High. "We need to perform further studies to translate this finding into safe, effective treatments for hemophilia and other single-gene diseases in humans, but this is a promising strategy for gene therapy." She continued, "The clinical translation of genetic therapies from mouse models to humans has been a lengthy process, nearly two decades, but we are now seeing positive results in a range of diseases from inherited retinal disorders to hemophilia. In vivo genome editing will require time to mature as a therapeutic, but it represents the next goal in the development of genetic therapies."


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Contact: John Ascenzi
ascenzi@email.chop.edu
267-426-6055
Children's Hospital of Philadelphia
Source:Eurekalert  

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