Guide RNA arrays steer base editors to specific loci, enabling dense plant mutagenesis without double-strand breaks or major off-target effects.
Chemically modified C9orf72 siRNA improves selective CNS gene silencing for ALS and FTD while addressing delivery and off-target constraints.
Sequence-specific siRNA silences CD33 mRNA to lower Siglec3 levels and modulate neuroinflammation in CNS-targeted treatment.