Nanoparticle-encapsulated siRNAs for gene silencing in the haematopoietic stem-cell niche

Nat Biomed Eng. 2020 Nov;4(11):1076-1089. doi: 10.1038/s41551-020-00623-7. Epub 2020 Oct 5.

Abstract

Bone-marrow endothelial cells in the haematopoietic stem-cell niche form a network of blood vessels that regulates blood-cell traffic as well as the maintenance and function of haematopoietic stem and progenitor cells. Here, we report the design and in vivo performance of systemically injected lipid-polymer nanoparticles encapsulating small interfering RNA (siRNA), for the silencing of genes in bone-marrow endothelial cells. In mice, nanoparticles encapsulating siRNA sequences targeting the proteins stromal-derived factor 1 (Sdf1) or monocyte chemotactic protein 1 (Mcp1) enhanced (when silencing Sdf1) or inhibited (when silencing Mcp1) the release of stem and progenitor cells and of leukocytes from the bone marrow. In a mouse model of myocardial infarction, nanoparticle-mediated inhibition of cell release from the haematopoietic niche via Mcp1 silencing reduced leukocytes in the diseased heart, improved healing after infarction and attenuated heart failure. Nanoparticle-mediated RNA interference in the haematopoietic niche could be used to investigate haematopoietic processes for therapeutic applications in cancer, infection and cardiovascular disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bone Marrow Cells / drug effects
  • Bone Marrow Cells / metabolism
  • Cells, Cultured
  • Disease Models, Animal
  • Drug Delivery Systems / methods*
  • Endothelial Cells / drug effects
  • Endothelial Cells / metabolism
  • Gene Silencing / drug effects*
  • Hematopoietic Stem Cells / drug effects*
  • Hematopoietic Stem Cells / metabolism
  • Mice, Inbred C57BL
  • Myocardial Infarction / prevention & control
  • Nanoparticles / administration & dosage*
  • Nanoparticles / chemistry*
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry*
  • Stem Cell Niche / genetics*

Substances

  • RNA, Small Interfering