Nanomedicine has transformed therapeutic strategies by enabling precise delivery of nucleic acid-based drugs, including small interfering RNA (siRNA), messenger RNA (mRNA), and antisense oligonucleotides. A landmark achievement is Onpattro (patisiran), the first FDA-approved RNAi therapy, which employs lipid nanoparticles (LNPs) to silence transthyretin in hereditary amyloidosis. Its approval validates RNAi as a viable therapeutic modality and underscores the central role of nanocarriers in clinical translation. Despite this success, barriers such as nanoparticle stability, targeted delivery, immunogenicity, and manufacturing scalability remain. Recent advances in mRNA vaccines, CRISPR-based gene editing, and stimuli-responsive nanoparticles are addressing these challenges, supported by growing clinical case studies and real-world data. This review highlights Onpattro’s clinical development, compares delivery platforms, discusses translational challenges, and examines emerging technologies that will guide the next generation of RNAi nanomedicines in personalized therapy.
Dilpreet Singh, Satvir Singh, Nitin Tandon et al.· Iranian Journal of Basic Med...· 1 citation
BACKGROUND
Diabetic burn wounds are challenging comorbid conditions to heal, hence shikonin, a traditional phytoconstituent was nanofabricated in the present study. This study is the continuation of our previously published work including shikonin-loaded nanoemulsion against wound pathogens.
OBJECTIVE
Current work aims to further prepare electrospun scaffolds to promote wounded skin regeneration.
METHODS
Studies including DLS, ATR, XRD, SEM were used to assess pharmaceutical stability of system. In vivo model, western blotting (protein expression of MMP-9), soluble collagen and MMP-9 ELISA kit assays, molecular docking and simulations studies were used to evaluate pharmacodynamic stability.
RESULTS
The developed scaffolds exhibited chemical stability, improved aqueous solubility with average diameter of 235 ± 23 nm. The wound area got remarkably decreased (4.923 ± 0.935%) with high collagen content (88.515 ± 2.23%) and lower MMP-9 content (6.726 ± 0.820%) than the comparable groups.
CONCLUSION
The present study displayed the developed system with remarkable wound healing potential.
Kirandeep Kaur, A. Monga, Anshula Mehra et al.· Journal of Microencapsulatio...· 0 citations
