abstract |
The present invention relates to antisense oligonucleotides capable of modulating the expression of ATXN2 in a target cell. The oligonucleotide hybridizes to ATXN2 mRNA. The invention further relates to conjugates and pharmaceutical compositions of said oligonucleotides, and methods of using said oligonucleotides for treating neurodegenerative diseases such as spinocerebellar ataxia type 2 (SCA2), Amyotrophic Lateral Sclerosis (ALS), alzheimer's disease frontotemporal dementia (FTD), parkinson's syndrome, and disorders with TDP-43 proteinopathies. |