ABSTRACT
Gene and enzyme replacement therapies are the most sophisticated, cutting-edge technologies available to treat genetic diseases (Rader, 1997; Yogalingam et al., 1997; Kozarsky et al., 1996; Vomdahl et al., 1998; O’Connor et al., 1998; Gornati et al., 1998; Kikuchi et al., 1998). These “high-tech” approaches are challenging, multi-faceted, specific, and innovative, but they are also researchintensive, costly, and restrictive. It is unlikely that most genetic diseases will be treated this way in the near future, especially orphan diseases with only a few hundred or a few thousand cases. There is a “low-tech” therapy that relies on dietary manipulation.
