🔬 Nature-Inspired Nanotechnology for RNA Delivery to Myeloid Cells & Bone Marrow Progenitors
🚀 A breakthrough in nucleic acid therapeutics! Published in Nature Nanotechnology, this study unveils a lipoprotein-based nanotechnology platform for precise RNA delivery to myeloid cells and hematopoietic stem and progenitor cells (HSPCs) in the bone marrow—a longstanding challenge in gene therapy.
🔹 Why It Matters?
🔬 Overcoming stability & targeting hurdles: Delivering RNA therapeutics (siRNA, ASOs, mRNA) systemically remains challenging due to degradation and non-specific uptake.
💡 Nature-inspired approach: The study utilizes apolipoprotein nanoparticles (aNPs) that mimic HDL (high-density lipoproteins) to stably incorporate RNA payloads for immune and bone marrow cell targeting.
🩸 Clinical potential: Enables gene silencing, protein expression, and immune modulation for treating genetic, inflammatory, and immune-related diseases.
🔹 Key Findings:
🧪 Library of aNP formulations developed & characterized for optimized RNA delivery.
🛠aNP-siRNA successfully silenced Lamp1 expression in immune cells after IV administration.
🎯 Gene regulation demonstrated in myeloid & HSPCs, outperforming standard lipid nanoparticles (LNPs).
🦠Therapeutic validation: Effective gene silencing in a murine tumor model showcases translational potential.
💊 Versatility: aNPs also support splice-switching with ASOs & mRNA-based protein production in bone marrow progenitors.
🔹 The Future of RNA-Based Therapies
This nature-inspired RNA delivery platform paves the way for targeted immunoregulation, next-gen vaccination strategies, and gene editing for hereditary disorders—with high biocompatibility, modular design, and scalable production making it a strong candidate for clinical translation.
🔗 Read more: Nature NanotechnologyÂ
💡 What are your thoughts on nature-inspired drug delivery for RNA-based therapies? Let’s discuss! 👇