Lariat RNA structures resist serum nucleases and maintain 70% potency after seven days, solving rapid degradation in traditional siRNA therapies.
Internal spacers in an 18-mer RNA interference agent improve nuclease resistance and activity duration compared to standard siRNA formats.
Cholesterol-conjugated double-stranded RNA agents silence SGLT2 transcripts while resisting nuclease degradation in vivo.
LNA gapmers hybridize with DUX4 mRNA to suppress expression, addressing the lack of curative options for Facioscapulohumeral Muscular Dystrophy.
Lipophilic conjugated oligonucleotides inhibit specific microRNAs while resisting nucleolytic degradation during systemic circulation.
GalNAc-conjugated RNAi compositions silence C3 transcripts, avoiding invasive procedures and side effects like cataracts.
DNase I digestion removes weak binders during single-cycle selection, preserving sequence diversity and reducing process time compared to multi-round SELEX.
Modified oligonucleotides target nuclear lncRNAs to displace PRC2, resolving inefficiency in regulating gene expression for neurodegeneration therapies.
Circular ACE-tRNAs revert premature termination codons without ototoxicity or missense mutations.
Sb-TIP2-3 promoter sequences drive tissue-specific expression in plant roots, resolving the contradiction between continuous activity and spatial control.
RNA fragment with stem-loop structure induces defense gene expression to prevent bacterial resistance in plants.
Experimental screening of candidate sequences resolves the contradiction between rapid computational design and reliable inhibitory effectiveness.
siRNA silences HNF4α-P2 isoforms to treat liver failure, addressing ineffective conventional therapies that fail to improve hepatocyte function.
TREMs pair with mutant codons to increase isoacceptor tRNA abundance, restoring protein translation efficiency despite genetic variations.
Circular Tau-TfR bispecific aptamers cross the blood-brain barrier via transferrin receptor-mediated transcytosis, reducing Tau protein levels in brain tissue.
Segmented siRNA strands self-assemble into double-stranded structures with 3' overhangs to enhance stability and inhibit target mRNA levels.
Recombinant nucleic acid molecules form hairpin structures with specific mismatches to enhance RISC incorporation and gene knockdown.
dsRNA agents silence KISS1 mRNA via RISC cleavage, restoring insulin secretion despite elevated glucagon levels in type 2 diabetes.
Modified oligonucleotides bind complementary FUS RNA sequences to reduce protein levels, addressing ineffective current therapies for ALS and FTLD.
Modified morpholino dsRNA resists nuclease degradation while maintaining sequence specificity through cationic backbone adjustments.
Modified nucleotides enhance stability and binding affinity to degrade PMS1 mRNA, addressing insufficient silencing in trinucleotide repeat disorders.
Phosphorothioate and phosphodiester linkages in oligomeric compounds improve tolerability while maintaining splicing modulation activity.
Removing hydrogen bond acceptors at position 6 of purine nucleobases prevents AMPA receptor antagonism while maintaining therapeutic efficacy.
Composite modified antisense oligonucleotides induce dystrophin exon 53 skipping to overcome limited efficacy in Duchenne muscular dystrophy treatment.
Conjugating peptide ligands to iRNA agents overcomes physiological barriers to deliver therapeutic RNA into the central nervous system and ocular tissues.
Targeting CHiLL1 and CHiLL2 lncRNAs with nucleic acid agents overcomes platinum drug resistance in KRAS-mutated non-small-cell lung cancer.
NR2F6 inhibition suppresses pathological angiogenesis via RNA interference, preventing tumor endothelial drug resistance.
Dual-specific aptamers bind HER2 and CD16 markers to induce cancer cell lysis while reducing side effects from traditional monoclonal antibodies.
Target mTOR and Akt pathways with RNA interference to resolve persistence limits in aquaculture growth promotion.
miR-23a-3p and miR-27a-3p mimics reduce Puma, Noxa, and Bax activation to limit cortical lesion volume following traumatic brain injury.
Fluorescent aptamers bind ovarian cancer cells to resolve low specificity in early-stage diagnosis.
Antisense inhibitors block microRNA binding to the SERPINC1 gene, increasing antithrombin production.
A replication-competent adeno-associated virus vector encodes an RNAi molecule to inhibit helper virus genes.
A nutritional composition containing miR-3141 modulates Claudin-2 and SLC2A1 gene expression to enhance infant gut barrier function.
Ethylene glycol modifications on oligonucleotides enable robust or modest target mRNA silencing across varying time periods.
Sequence-specific LNA ASOs target telomeric RNA precursors to impair maintenance in ALT cells without selecting resistant clones.
Stem-loop DNA aptamer binds L-ascorbic acid, preventing oxidation and preserving antioxidant function.
HIRA inhibitors disrupt viral DNA chromatinization, eliminating persistent cccDNA reservoirs that sustain chronic hepatitis B infection.
A nutritional composition containing miR-3184 modulates gene expression to support infant growth.
Oligomeric compounds reduce PTBP1 expression to promote neuronal differentiation, addressing limited neurogenesis options in neurodegenerative diseases.