Usually, there are helper proteins, called “chaperones,” that work throughout the cell to make sure other proteins fold correctly and prevent them from clumping. However, in diseases like HD, this ...
To better understand how the MutSβ complex can make the DNA zipper worse, the authors used a technique called cryo-electron ...
SOM3355 is still an investigational drug, and only a Phase 3 trial can determine whether it is safe and effective enough to seek approval. But the combined momentum from the EMA and FDA reflects a ...
Scientists discover a rare genetic quirk that may extend the age of onset in people with Huntington’s disease by boosting cellular cleanup.
In recent years, HD research headlines have trended toward huntingtin lowering: experimental therapies that target the root genetic cause of HD. But there are also several drugs in development to ...
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger ...
People who develop Huntington’s disease (HD) are born with the genetic change that causes the disease. So why does it take decades, usually around 40 to 50 years, for the symptoms of the disease to ...
Last month, we relayed positive news from uniQure’s trial testing AMT-130, a gene therapy delivered via brain surgery to lower huntingtin (HTT). Data released by uniQure in June suggested AMT-130 was ...
UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the ...
For those who were following the live tweets from HDBuzz about the CHDI HD Therapeutics Conference or tuned in to the HDSA Convention, we may have caught your attention with the new HD staging system.
The gene MSH3 is getting a lot of attention in HD research lately. New findings suggest MSH3 lowering could halt CAG repeat expansions, offering a new therapeutic avenue. One of the most interesting ...
CRISPR is short for “clustered regularly interspaced short palindromic repeats” – quite a mouthful! That’s essentially just science-speak for short strings of DNA letters that break up repeating parts ...
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