Muscle-targeting antibodies use receptor-mediated internalization to deliver oligonucleotides and support dystrophin exon skipping in muscle cells.
DNA- or RNA-delivered aptamers bind toxic metals before absorption, helping reduce reliance on chelation therapy after cumulative exposure.
Excess PCSK9 can elevate LDL cholesterol; guide RNA and Cas9 delivered in lipid nanoparticles target the gene to reduce its expression.
Targeted dsRNA silences excess MUC5B associated with rs35705950-T, addressing fibrosis progression while preserving upper-airway infection protection.
Sequential esterification, substitution, hydrogenation, protection, and hydrolysis produce a lipid for targeted nucleic acid delivery.
Exosome carriers deliver HBB DNA and rs334-targeting siRNA to address sickle cell mutations without viral-vector integration or chemotherapy.
Nucleic acids, expression vectors, and antisense oligonucleotides adjust L1 RNA activity to support bone mass or reduce aging-linked cellular activity.
Controlled surface density of TLR 7/8 and 9 agonists concentrates immune stimulation on nanoscale constructs, improving response while reducing systemic toxicity.
Stem II loop sequences and a position-7 uracil improve hammerhead ribozyme turnover for more efficient target RNA cleavage.
Sequence-specific siRNA silences the C1858T PTPN22 allele while sparing wild-type function and reducing IL-2 production in T lymphocytes.