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Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

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Pharmacological or Molecular Chaperones are small molecules that are able to cross the blood brain barrier into the central nervous system. These chaperones attach to an inactive enzyme so that it takes the correct functional shape. However, chaperones only work with certain mutations, and have the potential to reduce enzyme function if given at the wrong dose

 

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