splice-switching oligonucleotides

Splice-switching oligonucleotides (SSOs) are short, synthetic strands of nucleic acids designed to modify how genes are expressed. They work by binding to specific regions of pre-messenger RNA (pre-mRNA), the precursor to messenger RNA (mRNA). This binding influences the cell’s splicing process, which normally edits pre-mRNA to produce functional proteins. By redirecting splicing, SSOs can restore or alter the production of proteins, potentially correcting genetic mutations or controlling disease-related gene expression. They are a promising approach for treating certain genetic disorders by precisely modifying gene messages without changing the DNA itself.