Repair and regeneration of tissues using endogenous stem cells represent an ultimate goal in regenerative medicine. To our knowledge, human lens regeneration has not yet been demonstrated. Currently, the only treatment for cataracts, the leading cause of blindness worldwide, is to extract the cataractous lens and implant an artificial intraocular lens. However, this procedure may pose notable risks of complications. In this paper we isolate lens epithelial stem/progenitor cells (LECs) in mammals and show that Pax6 and Bmi1 are required for LEC renewal. We design a surgical method for removal of cataract that preserves endogenous LECs and achieves functional regeneration of lens in rabbits and macaques, as well as in human infants with cataracts. Our method differs conceptually from the current practice, preserves endogenous LECs and their natural environment maximally, and regenerates lenses with visual function. Our approach demonstrates a novel treatment strategy for cataracts and provides a new paradigm for tissue regeneration using endogenous stem cells.

endogenous stem cells  /  lens regeneration  /  pediatric cataract