Myotonic Dystrophy type 1 is a genetic neuromuscular disorder caused by a mutation in the DMPK gene, resulting in a trinucleotide (CUG) repeat expansion in the expressed RNA. Our DM1 program targets and destroys the toxic CUG repeats.

Huntington’s Disease is a genetic neurodegenerative disorder caused by a mutation in the HTT gene, resulting in a trinucleotide (CAG) repeat expansion in the expressed RNA. Our Huntington’s Disease program targets and destroys the excessive CAG repeats.

Spinocerebellar Ataxia Type 1 (SCA1) is a genetic neurodegenerative disorder caused by a mutation in the ATXN1 gene, resulting in a trinucleotide (CAG) repeat expansion in the expressed RNA. Our SCA1 program targets and destroys the excessive CAG repeats.

C9orf72-related Amyotrophic Lateral Sclerosis (ALS) is a genetic motor neuron disorder caused by a mutation in the C9orf72 gene, resulting in hexanucleotide (G4C2 and C4G2) repeat expansions. Our C9orf72-ALS program targets and destroys the hexanucleotide repeats.

C9orf72-related Frontotemporal dementia (FTD) is a genetic neurodegenerative disorder caused by a mutation in the C9orf72 gene, resulting in hexanucleotide (G4C2/C4G2) repeat expansions. Our C9orf72-FTD program targets and destroys the hexanucleotide repeats.