Novel Methods for Generating Retinal Pigment Epithelium Cells from Induced Pluripotent Stem Cells

High efficiency methods for producing retinal pigment epithelial cells (RPE) from induced pluripotent stem cells (iPSCs) are disclosed. The RPE is a polarized monolayer in the vertebrate eye that separates the neural retina from the choroid, and performs a crucial role in retinal physiology by forming a blood-retinal barrier and closely interacting with photoreceptors to maintain visual function. Many ophthalmic diseases, such as age-related macular degeneration, are associated with a degeneration or deterioration of the RPE. The iPSCs are produced from somatic cells, including retinal pigment epithelial cells, such as fetal RPE. These methods involve producing embryoid bodies from human iPSCs, culturing the embryoid bodies using specific media to induce differentiation into RPE and growing the differentiated RPE cells in a defined media to generate human RPE cells. The investigators also developed methods for detecting RPE cells and authenticating RPE cells; determining agents that can affect the production of RPE cells from an iPSC; and identifying an agent that can increase RPE survival in response to a proteo toxic insult or stress. The novel methods and RPE cells disclosed here can be useful for both pre-clinical and clinical studies involving RPE.

Potential Commercial Applications: Competitive Advantages:
The methods described here can be used to:
  • produce RPE cells for use in screening for novel ocular therapeutics and for identifying toxic side effects of drugs
  • produce RPE cells for use in novel cell-based therapies
  • produce cells to study pathophysiology of RPE
 The methods described here:
  • dramatically increase the efficiency of iPSC differentiation into RPE
  • produce superior quality RPE
  • produce RPE cells that are fully authenticated
  • provide ways to perform high throughput screens with RPE cells

Development Stage:
  • Prototype
  • Early-stage
  • In vitro data available


Kapil Bharti (NEI)  ➽ more inventions...

Sheldon Miller (NEI)  ➽ more inventions...

Intellectual Property:
US Application No. 14/764,959
US Application No. 61/759,988
PCT Application No. PCT/US2014/014160

Collaboration Opportunity:

The National Eye Institute is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate or commercialize iPSC to RPE differentiation protocol, its clinical, screening, and translational applications. For collaboration opportunities, please contact Alan Hubbs, Ph.D. at Click here to view the NEI collaborative opportunity announcement.

Licensing Contact:
Edward (Tedd) Fenn, J.D.
Phone: 240-276-6833

OTT Reference No: E-251-2012/3
Updated: May 31, 2013