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

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

and other trinucleotide repeat disorders, because such changes in DNA occur naturally in people without disease or who only have mild symptoms. “Not only does this study show for the first time ...

PepGen to focus on development of DM1 programme following 10 mg/kg PGN-EDO51 update: Boston Friday, May 30, 2025, 14:00 Hrs [IST] PepGen Inc, a clinical-stage biotechnology compan ...

3 Sobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology ... to the genetic testing for HD causative mutations, that is, IT15 gene trinucleotide-repeat expansion.

The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.