In people who have the condition, which typically starts causing motor and cognitive symptoms in middle age, mutant huntingtin protein (HTT) clumps inside neurons and invades their nuclei—processes ...

Huntington's disease is a devastating neurogenerative disorder caused by a mutation in the HTT gene that results in cells producing abnormally large versions of the huntingtin protein. These ...

The resulting mutant HTT protein elicits many toxic downstream effects, particularly in the central nervous system (CNS), leading to the gradual loss of neurons, failing motor skills and ...

HD is caused by an expanded glutamine repeat in the N-terminal region of the huntingtin (HTT) protein. The disease onset has distinct patterns in juvenile and adult patients, but with similar disease ...

SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology. "We are excited to present on our Huntingtin-lowering ...

"Our unique approach to selectively target the mHTT protein while preserving wild-type is a clear example ... biodistribution and allele selective editing and reduction of mutant HTT protein in ...