Scientists at Cincinnati's Children's Hospital Medical Center report that they may have discovered a new approach to molecular gene therapy and a much-needed improved treatment option for children with Hurler's syndrome.
The study released by the research team at the Cincinnati Children's Hospital Medical Center is also the first to demonstrate that developing red blood cells can be used to produce lysosomal enzymes. The lack of this enzyme in children with Hurler syndrome causes progressive tissue damage to organs and the central nervous system which often results in early death.
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