Huntingtin’s Roles Outside of Huntington’s Disease The ... For now, the evidence for a causal connection between disruption in normal protein function and the onset of neurodegenerative disease is ...

or huntingtin in Huntington’s. But when not mutated, misfolded, or otherwise misbehaving, these proteins seem to play critical roles in brain development and function, leading some researchers to ...

such as some DNA segments of the huntingtin gene, which codes for a protein important for many neurological functions. When an APOBEC deaminates parts of that gene containing the CAG/CTG sequence ...

A longstanding enigma in the Huntington's disease is how the mutated protein derived from the huntingtin gene is ... from yeast to human, but its function in yeast remains unknown.

Huntingtin was the first disease gene mapped ... and a decline in cognitive function. Interestingly, J. Hoffman first reported a juvenile form of HD in 1888 through his studies of a three ...

These expanded huntingtin proteins aggregate inside cells and damage ... in the nuclear envelope that compromise its barrier ...

Aggregation of huntingtin protein with an expanded polyglutamine region is enhanced by its 17-residue N-terminal domain, which binds to itself and to the polyglutamine region. This enhancement is ...

Huntington's disease is a genetic condition caused by an unusually long DNA sequence (CAG) in the huntingtin gene ... studies suggest that loss of MSH3 function is relatively well-tolerated ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT ... while preserving wild-type protein, which is essential for cellular functions and physiological health.

In preclinical studies, LETI-101 has demonstrated selective reduction of disease-causing mutant huntingtin (mHTT ... model (BACHD), while preserving wild-type protein, which is essential for cellular ...