News Finding: We conclude that this #ZFN-based platform for expression of therapeutic proteins from the albumin locus is a promising approach for treatment of #MPS II and other #lysosomal diseases.
  $SGMO #raredisease  
Paper Finding: Remarkably, the high level of IDS expression achieved in animals administered the highest dose of ZFN+hIDS donor encoding vectors prevented emergence of a neurocognitive deficit exhibited in control MPS II animals.
News Context: We conclude that this #ZFN-based platform for expression of therapeutic proteins from the albumin locus is a promising approach for treatment of #MPS II and other #lysosomal diseases.
  $SGMO #raredisease  
Paper Context: The levels of hIDS achieved in the circulation increased in a dose-dependent manner, and IDS was subsequently taken up from the circulation by secondary tissues, resulting in normalization of GAG storage levels. Remarkably, the high level of IDS expression achieved in animals administered the highest dose of ZFN+hIDS donor encoding vectors prevented emergence of a neurocognitive deficit exhibited in control MPS II animals. 14, However, episomal expression of a transgene in the liver is unlikely to achieve long-term expression in a pediatric patient population, due to the high proliferative rate of hepatocytes in this population.