Aniridia is a devastating ocular disease requiring intensive eye care, social and community support from birth and throughout an individual’s lifetime.

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WG3: Stem Cells and Regenerative Medicine

WG3: Stem Cells and Regenerative Medicine

Approaches of stem cell biology, tissue engineering, gene delivery (eg. viral vectors), gene modification (CRISPR/Cas9 technology) or gene modulation (through miRNA) for developing translational regenerative therapeutic strategies for the treatment of aniridia.


  • Identify approaches to improve current stem cell transplantation.
  • Test new approaches of tissue engineering/gene delivery in vitro.
  • Test gene modulation/editing specifically for PAX6.
  • Explore use of iPSC and MSC technology for replacing limbal stem cells.


Limbal stem cells colonies stained with crystal violet.

  • Report on methods to improve limbal stem cell survival after transplantation.
  • Report on progress of gene/stem cell/tissue engineering approaches for PAX6 haploinsufficiency.

WG3 Leader: Dr Stefano FERRARI (