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

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

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 ...

CRISPR–Cas base editing of trinucleotide repeats shows promise in reducing somatic repeat expansions in Huntington’s disease and Friedreich’s ataxia, offering a potential new therapeutic ...

Autosomal recessive ataxias may present with chorea, but generally onset is in the first two decades. Friedreich ataxia (FA) is by far the most common of these forms, occurring in about 1 in 50 000 ...

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 ...