ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...
This 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 ...