Lipid nanoparticles for bone marrow-targeted RNA therapeutics
📄 英文摘要 English Abstract
Lipid nanoparticle (LNP)-mediated delivery of therapeutic RNA molecules to the bone marrow (BM) holds transformative potential to treat hematological malignancies such as leukemia and for advancing immune and regenerative therapies. Despite this promise, LNPs exhibit poor BM accumulation following systemic administration. Formidable physiological barriers including rapid clearance by the mononuclear phagocyte system, predominant off-target sequestration in the liver, complement activation and inefficient endosomal escape currently limit their efficacy for extrahepatic applications. This review characterizes the complex BM physiology and the multifaceted challenges hindering LNP delivery to this organ. We discuss successful in vivo passive and active targeting strategies, evaluate emerging preclinical in vitro models and highlight innovative LNP design strategies aimed at enhancing particle stability and circulation time. Together, these advances establish a framework for the next-generation of RNA-therapeutics dedicated to treating BM-associated diseases.
📄 中文摘要 Chinese Abstract
📋 英文结构化总结 English Structured Summary
摘要整理
Background:
Lipid nanoparticle (LNP)-mediated delivery of therapeutic RNA molecules to the bone marrow (BM) holds transformative potential to treat hematological malignancies such as leukemia and for advancing immune and regenerative therapies. Parabiotics (also termed paraprobiotics) are defined as non-viable microbial cells or their components, including peptidog.
Methods:
N/A - not provided in the abstract.
Results:
N/A - not provided in the abstract.
Data Summary:
N/A - no quantitative results or key statistics provided.
Conclusions:
N/A - not provided in the abstract.
Practical Significance:
The abstract points to therapeutic potential for LNP-mediated delivery of therapeutic RNA molecules to the bone marrow (BM) in hematological malignancies such as leukemia and for advancing immune and regenerative therapies, and for parabiotics as next-generation microbiome therapeutics.
📋 中文结构化总结 Chinese Structured Summary
背景:
脂质纳米颗粒（LNP）介导的治疗性RNA分子递送至骨髓（BM）具有变革性潜力，可用于治疗白血病等血液系统恶性肿瘤，并推动免疫和再生疗法的发展。副益生菌（也称为旁益生菌）被定义为非活性微生物细胞或其成分，包括肽聚糖。
方法:
不适用——摘要中未提供。
结果:
不适用——摘要中未提供。
数据摘要:
不适用——未提供定量结果或关键统计数据。
结论:
不适用——摘要中未提供。
实践意义:
摘要指出，脂质纳米颗粒（LNP）介导的治疗性RNA分子递送至骨髓（BM）在白血病等血液系统恶性肿瘤中具有治疗潜力，并有助于推动免疫和再生疗法的发展；同时，副益生菌可作为下一代微生物组疗法。