Sequence-specific removal of mutant transcripts

Sequence-specific removal of mutant transcripts refers to molecular strategies that selectively target and eliminate RNA molecules containing specific mutations, while sparing the wild-type (non-mutant) transcripts. This approach is crucial for correcting or mitigating the effects of disease-causing mutations at the RNA level, especially in genetic disorders where mutant mRNA leads to production of dysfunctional or toxic proteins. Technologies used for this purpose include antisense oligonucleotides (ASOs), RNA interference (RNAi), and CRISPR-based methods. This is particularly valuable in dominant-negative diseases and allows allele-selective therapy.

Targeted degradation or silencing of mutant mRNA via antisense oligonucleotides, RNA interference, or CRISPR interference.