Source: Cincinnati Children's Hospital Medical Center
Date: November 6, 2009
Summary:
Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme -preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome. The research team from Cincinnati Children's Hospital Medical Center reports its preclinical laboratory results this week in the early edition of Proceedings of the National Academy of Sciences.
The study suggests a new approach to molecular gene therapy and a much-needed improved treatment option for children with Hurler's syndrome, said Dao Pan, Ph.D., a researcher in the Division of Experimental Hematology/Cancer Biology at Cincinnati Children's and the study's principal author. It also is the first study to demonstrate that
developing red blood cells can be used to produce lysosomal enzymes.
