Publications by authors named "M-K Place"
Mol Ther Nucleic Acids
March 2024
Article Synopsis
- Antisense oligonucleotides (ASOs) were the first to successfully target and reduce mutant SOD1 expression in amyotrophic lateral sclerosis (ALS) therapy.
- Short interfering RNAs (siRNAs) also offer a gene-silencing mechanism but faced delivery challenges to the central nervous system (CNS).
- The study introduces a new siRNA-ACO conjugate that enhances delivery to CNS tissues, showing promising results in slowing ALS progression and improving motor function in SOD1 mice.
