Children of a parent with HD have a 50% chance of receiving the mutated HTT gene and thus developing the disease later in life. Today, through the use of PCR-based techniques, individuals at risk ...

引言在人类基因组的浩瀚密码中,一段名为"Huntingtin(HTT)"的基因序列暗藏杀机。当这个基因上出现过多CAG三核苷酸重复时,就会引发被称为"亨廷顿舞蹈症(Huntington's ...

Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by a polyglutamine repeat expansion within the huntingtin protein. HD is characterized by problems with ...

Huntington’s disease is an inherited neurological disorder causing movement, cognitive, and behavioral issues. Effective ...

HDBuzz reported live updates on Bluesky from the 2025 HD Therapeutics Conference. Read on for coverage of Day 1. #CHDI2025 ...

A new discovery offers hope for Huntington’s disease. This discovery provides hope that a DNA repair process may help slow or ...

In 1993, researchers discovered that the disease is caused by an expanded stretch of CAGs in the HTT gene. Most people inherit versions of the gene with 15 to 35 consecutive CAGs and never develop ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...

The company’s second development candidate, ATL-101, is a di-siRNA designed to silence the HTT gene for the treatment of Huntington’s disease. Huntington’s disease is a progressive ...

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 ...