Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and ...

About DT-216P2 DT-216P2 is an improved formulation of a GeneTAC ® small molecule designed to specifically target the GAA repeat expansion mutation that is the underlying cause of FA and restore the ...

Early research shows that editing expanding trinucleotide repeats halts the lengthening process that causes neurological disease.

In people with Friedreich’s ataxia, this sequence may repeat 66 to over 1,000 times, according to U.S. National Library of Medicine’s Genetics Home Reference. When production of this DNA ...

Friedreich ataxia (FA) is a rare ... 3 Most patients with FA are homozygous for the GAA repeat expansion mutation. Larger nucleotide expansions correlate with earlier age of onset and more ...

There are many types, including cerebellar ataxia, sensory ataxia, vestibular ataxia, Friedreich’s ataxia and more general forms of hereditary ataxias. The most common symptoms of the disease ...

And perhaps the most useful such handle yet found is the phenomenon called trinucleotide repeat expansion. This occurs when any three of the four nucleotide subunits in DNA material begin to ...

Friedreich’s ataxia (FA) is a neurodegenerative disease most people have never heard of, and it remains widely misunderstood —and for the hundreds of Canadians who live with it, the lack of ...

What Is Friedreich’s Ataxia? Friedreich’s ataxia (FA) is a rare condition. It causes damage to your nervous system that gets worse over time. As a result, you may have abnormal movements ...

However, the team thinks that a therapeutic strategy that introduces interruptions into repeat tracts could one day help treat Huntington’s, Friedreich’s ataxia, and other trinucleotide repeat ...