Scientists discover a rare genetic quirk that may extend the age of onset in people with Huntington’s disease by boosting ...
To better understand how the MutSβ complex can make the DNA zipper worse, the authors used a technique called cryo-electron ...
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 ...
The new twist in this latest study from Brazil is where the stem cells come from. Researchers used human dental pulp stem cells, collected from the soft tissue inside teeth, to create an experimental ...
Huntington’s disease (HD) develops when people have a repetition of the genetic letters C-A-G in their huntingtin gene. People with 40 or more repeats will develop the disease during their lifetime, ...
A BLA, or Biologics Licensing Application, is the formal dossier of information that a company uses to request permission to market a biologic, which is a therapy created from living organisms, like ...
Data from the HART study of Huntexil for HD symptoms now published, but we still need a new, larger trial ...
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 ...
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 ...
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 ...
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