Inflammation-responsive framework nucleic acid-enabled effective intestinal accumulation for ulcerative colitis therapy - PubMed
3 hours ago
- #Ulcerative colitis
- #DNAzyme
- #Inflammation
- Therapeutic oligonucleotides targeting GATA3 show promise for ulcerative colitis (UC) treatment.
- Tetrahedral framework nucleic acids (tFNAs) enable effective intravenous delivery of GATA3-targeted oligonucleotide (hgd40 DNAzyme) to the intestine.
- The hgd40 DNAzyme is assembled into tFNAs via an inflammation-responsive DNA duplex, allowing selective release in inflamed cells via APE1.
- hgd40-tFNA demonstrates effective intestinal accumulation and selective knockdown of pathogenic genes in inflamed cells.
- In DSS-induced colitis mice, hgd40-tFNA mitigates weight loss and colonic shortening, restoring intestinal epithelial homeostasis.
- tFNAs highlight potential for intravenous administration in treating inflammatory intestinal diseases.