CD133 progenitor cells from the bone marrow contribute to retinal pigment epithelium repair

Stem Cells. 2009 Feb;27(2):457-66. doi: 10.1634/stemcells.2008-0836.


Our goal was to define a clinically significant population of cells by utilizing a single-step selection process to enrich hematopoietic cells capable of regenerating the retinal pigment epithelium (RPE). Utilizing intravitreal injection of bone marrow cells from a mouse with pigment (C57BL6:gfp) into albino recipient mice (C57BL6:Tyr(-)), we show that hematopoietic progenitor cells (HPCs) enriched for CD133 can regenerate RPE cells and improve retinal function. The chemokine CXCL12 (stromal cell-derived factor 1alpha) is essential for migration, incorporation, and RPE regeneration by CD133(+) HPCs. Once incorporated, CD133(+) HPCs become pigmented, adopt an RPE morphology, and express RPE-specific proteins, leading to partial functional recovery by electroretinogram. Human CD133(+) HPCs also incorporate in the retina and assume RPE morphology in nonobese diabetic/severe combined immunodeficient mice xenografts. These data show that a clinically accessible CD133(+) hematopoietic cell can home to an injured RPE layer, differentiate into cells with significant RPE morphology, and provide therapeutic functional recovery of the visual cycle.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • AC133 Antigen
  • Animals
  • Antigens, CD / metabolism*
  • Bone Marrow Cells / cytology*
  • Cells, Cultured
  • Enzyme-Linked Immunosorbent Assay
  • Flow Cytometry
  • Glycoproteins / metabolism*
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunohistochemistry
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Peptides / metabolism*
  • Pigment Epithelium of Eye / pathology*
  • Retinal Pigment Epithelium / pathology*
  • Stem Cells / cytology*
  • Stem Cells / metabolism*
  • Stem Cells / physiology


  • AC133 Antigen
  • Antigens, CD
  • Glycoproteins
  • PROM1 protein, human
  • Peptides
  • Prom1 protein, mouse