Long non-coding RNA delivery (lncRNA delivery)

Long non-coding RNA (lncRNA) delivery refers to the therapeutic modality of transporting lncRNA molecules or their inhibitors to target cells to modulate physiological or pathological pathways. lncRNAs are a heterogeneous class of transcripts longer than 200 nucleotides that do not encode proteins but play fundamental roles in gene regulation through scaffolding, acting as decoys for microRNAs, and guiding chromatin-modifying complexes [1][15][16]. Their dysregulation is a hallmark of many diseases, particularly cancer and cardiovascular disorders, making them highly attractive as therapeutic targets or agents for precision medicine [4][7]. Effective delivery remains a significant challenge due to the large molecular weight and instability of lncRNAs in biological fluids, requiring the use of advanced vectors such as lipid nanoparticles (LNPs), viral vectors, or exosome-based systems [1][6][8]. These delivery technologies are designed to protect the cargo from enzymatic degradation and facilitate intracellular entry and endosomal escape to allow the payload to reach its site of action in the cytoplasm or nucleus [3][13]. While lncRNA-based therapies like BC-819 have entered clinical investigation, the field must still overcome hurdles related to off-target effects, systemic toxicity, and immunogenicity to achieve widespread clinical application [5][9][11].

Other names

lncRNA-based therapeutics deliverylncRNA targetingLong non-coding RNA-mediated therapyRNA-based therapy delivery

lncRNA-based therapeutics function by delivering functional lncRNA mimics to restore tumor-suppressive activity or by using antisense oligonucleotides (ASOs) and siRNAs to induce the degradation of oncogenic lncRNAs, thereby modulating gene expression at epigenetic and transcriptional levels [1][5][10]. Delivery vehicles facilitate this by protecting the RNA from nucleases and enabling its passage across the plasma membrane and exit from endosomes [3][6].