ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...
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Surprising way that genetic mutation causes Huntington's disease changes understanding of the disorderThis type of "somatic expansion" occurs in only the specific types of brain cells that later die in Huntington's disease. Only once a cell's DNA expansion reaches a threshold number of CAGs ...
LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...
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