Embryonic stem cells help deliver 'good genes' in a model of inherited blood disorder
Sunday, February 13, 2011 - 16:10
in Biology & Nature
Researchers report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, beta-thalassemia. Some of the stem cell lines do not inherit the disease gene and can thus be used for transplantation-based treatments of the same mice. The findings could hold promise for a new treatment strategy for autosomal dominant diseases like certain forms of beta-thalassemia, tuberous sclerosis or Huntington's disease.