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Aptamer-functionalized lipid nanoparticles targeting osteoblasts as a novel RNA interference-based bone anabolic

Chao Liang1, Baosheng Guo2, Heng Wu3

  • 11] Institute for Advancing Translational Medicine in Bone &Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong SAR, China. [2] Institute of Basic Research in Clinical Medicine, China Academy of Chinese Medical Sciences, Beijing, China. [3] State Key Laboratory of Proteomics, Beijing Proteome Research Center, Beijing Institute of Radiation Medicine, Beijing, China. [4] Academician Chen Xinzi Workroom for Advancing Translational Medicine in Bone &Joint Diseases, Kunshan RNAi Institute, Kunshan Industrial Technology Research Institute, Kunshan, Jiangsu, China. [5] Institute of Integrated Bioinfomedicine &Translational Science, Hong Kong Baptist University Shenzhen Research Institute and Continuing Education, Shenzhen, China. [6] Shum Yiu Foon Shum Bik Chuen Memorial Centre for Cancer and Inflammation Research, Hong Kong Baptist University Shenzhen Research Institute and Continuing Education, Shenzhen, China. [7] Hong Kong Baptist University Branch of State Key Laboratory of Chemo/Biosensing and Chemometrics of Hunan University, Hong Kong, China. [8] Hong Kong Baptist University-Northwestern Polytechnical University Joint Research Centre for Translational Medicine on Musculoskeletal Health in Space, Shenzhen, China.

Nature Medicine
|February 10, 2015
PubMed

Insights

Researchers developed aptamer-functionalized lipid nanoparticles (LNPs) for targeted delivery of RNA interference (RNAi) to bone-forming cells. This novel strategy enhances bone formation and improves bone properties in rodents, addressing safety concerns of current bone anabolic therapies.

Area of Science:

  • Biotechnology
  • Regenerative Medicine
  • Molecular Biology

Background:

  • Current RNA interference (RNAi) bone anabolic strategies face safety and efficacy challenges due to the absence of osteoblast-specific delivery systems for osteogenic small interfering RNAs (siRNAs).
  • Targeted delivery of therapeutic agents to specific cell types is crucial for enhancing treatment efficacy and minimizing off-target effects.

Purpose of the Study:

  • To develop and evaluate osteoblast-specific aptamer-functionalized lipid nanoparticles (LNPs) for targeted delivery of osteogenic siRNA.
  • To assess the efficacy of this novel RNAi-based strategy in promoting bone formation and improving bone quality in vivo.

Main Methods:

  • Screening of aptamer CH6 using cell-SELEX for specific targeting of rat and human osteoblasts.
  • Development of CH6 aptamer-functionalized LNPs encapsulating pleckstrin homology domain-containing family O member 1 (Plekho1) siRNA (CH6-LNPs-siRNA).
  • In vitro assessment of osteoblast-selective uptake and in vivo evaluation of gene silencing, bone formation, microarchitecture, mass, and mechanical properties in osteopenic and healthy rodents.

Main Results:

  • CH6 facilitated selective uptake of Plekho1 siRNA by osteoblasts in vitro, primarily through macropinocytosis.
  • CH6-LNPs-siRNA achieved osteoblast-specific Plekho1 gene silencing in vivo.
  • The treatment promoted bone formation, enhanced bone microarchitecture, increased bone mass, and improved mechanical properties in both osteopenic and healthy rodents.

Conclusions:

  • Osteoblast-specific aptamer-functionalized LNPs represent a promising new strategy for RNAi-based bone anabolism.
  • This approach advances targeted siRNA delivery from the tissue level to the cellular level, improving selectivity and potentially safety.
  • The findings support the potential of this technology for treating bone loss conditions and enhancing bone regeneration.

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