Guide RNAs target heterozygous APOA1 SNPs to knock out the mutant allele while preserving functional apoA-1 expression and limiting amyloid fibrils.
Customized guide RNA directs CRISPR/Cas to the MYD88 L265P mutation, killing B-cell lymphoma cells while sparing healthy cells.
CRISPR guide RNAs selectively disable the mutant ELANE allele to treat SCN or CyN while preserving functional protein expression.
Selective RNA-targeting agents suppress toxic cytoplasmic G4R1 while preserving nuclear G4R1, reducing off-target cellular effects.
Chemically modified antisense oligonucleotides improve SNP-level target specificity while preserving effective nucleic acid modulation.
Selective siRNA silences the PLN R14del mutant transcript while preserving wild-type expression to improve cardiac function and lower arrhythmia risk.