Antibody-linked oligonucleotide complexes use receptor-mediated uptake in muscle cells to restore dystrophin expression more effectively.
A single dual-cassette AAV delivers mini-dystrophin and NF-κB/p65-shRNA to muscle, simplifying DMD therapy while reducing inflammation.
A single AAV CRISPR system deletes dystrophin exons 45-55 to restore functional expression in skeletal and cardiac muscle.
Cationic lipid particles balance endosomal escape with low cytotoxicity to protect nucleic acids and improve intracellular delivery.
Engineered Arc and endogenous Gag capsids encapsulate RNA cargo for more specific cellular delivery with fewer off-target effects.
Engineered AAV capsids use n-mer targeting motifs to boost muscle transduction, lower liver exposure, and enable lower-dose cargo delivery.
DPD-silencing shRNA inserted into HSV-CD boosts 5-FC to 5-FU therapy by limiting 5-FU breakdown and suppressing glioma metabolism.
GLP-1 ligand conjugates give oligonucleotides selective access to receptor-expressing cells, enabling targeted gene expression modulation.
Synthetic MSC-derived extracellular vesicles deliver anti-fibrotic microRNAs to modulate fibrogenic pathways and support tissue repair.
EpCAM-targeted aptamer-siRNA chimeras improve RNAi delivery to cancer cells beyond the liver, boosting efficacy while limiting side effects.
Engineered propagator cells raise phage titers to therapeutic levels by using receptor-matched host strains without requiring large culture volumes.
Serum-stable integrin ligands bind αvβ3 and αvβ5 to deliver RNAi cargo selectively into tumor and other integrin-expressing cells.
Blocking miR-379 and miR-541 together improves glucose and lipid metabolism in glucocorticoid-driven diabetes, obesity, and metabolic syndrome.
Dual vectors target the PCSK9 locus with nuclease-guided HDR to insert therapeutic genes and support durable expression in adult patients.
miRNA target sequences suppress transgene expression in HSCs and progenitors while preserving therapeutic activity in differentiated cells.
SNP-guided CRISPR cleavage introduces frameshift edits in mutant HTT, lowering mutHTT levels while sparing wild-type expression.
miR17-92 co-delivery suppresses GR pathway interference in AAV vectors, boosting mammalian cell transduction and transgene expression.
A dendritic linker with solid-phase synthesis and click chemistry raises aptamer-drug loading while preserving water solubility and conjugation efficiency.
A polymer-core exosome coating boosts RNAi loading, protects against degradation, and improves tumor cell uptake for scalable therapy.
Targeting the Staufen1-ATXN2 pathway helps restore autophagy, reduce toxic protein aggregates, and slow neurodegenerative decline.
A pH-responsive phospholipid stays neutral in body fluid but turns cationic for RNA loading, improving encapsulation while limiting cytotoxicity.
Modified Cpf1 effectors and compact delivery vectors improve target binding and editing preference while reducing off-target genome edits.
Specific sequence motifs direct miRNAs into exosomes or keep them in cells, improving targeted gene silencing for cancer and metabolic disease.
Tumor-homing auxotrophic bacteria deliver checkpoint RNAi locally to boost anti-tumor immunity while limiting systemic cytokine exposure.
Chemically modified antisense oligonucleotides improve ADAM33 mRNA silencing by RNase H cleavage, overcoming weak siRNA efficacy.
Dual anti-BAFFR and anti-BCMA antibody-siRNA conjugates suppress autoantibody production for longer-lasting myasthenia control.