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 ...

In Huntington's disease, a mutation in a protein called HTT causes nerve cells in the brain to break down over time. This can cause mood changes and balance problems, and eventually movement ...

SKY-0515 is Skyhawk's investigational small molecule RNA splicing modifier developed through the company's novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein ...

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 ...

LETI-101 combines Life Edit's proprietary CRISPR nuclease with AAV5 delivery to selectively target the T allele of an exonic SNP in the HTT gene to treat HD. In preclinical studies, LETI-101 has ...

LETI-101 combines Life Edit's proprietary CRISPR nuclease with AAV5 delivery to selectively target the T allele of an exonic SNP in the HTT gene to treat HD. In preclinical studies, LETI-101 has ...