Induced pluripotent stem cells and retinal degeneration treatment

  title={Induced pluripotent stem cells and retinal degeneration treatment},
  author={Jin Yang and Eva Nong and Stephen H. Tsang},
  journal={Expert Review of Ophthalmology},
  pages={5 - 8}
Lesions of the retinal pigment epithelium and photoreceptors lead to different kinds of retinal dystrophies, which are characterized by the grade of visual loss and the character of retinal atrophy. Some of these disorders are single-gene mutations, while others are polygenic with associated environmental factors. In the past, animal models, gene therapy and embryonic stem (ES) cells have been used to study and provide treatment. However, these methods have their limitations. Recently, the… 



Generation of retinal ganglion-like cells from reprogrammed mouse fibroblasts

  • N. Jindal
  • Biology, Medicine
    Annals of Neurosciences
  • 2011
Embryonic stem cells have been identified as a viable source of retinal progenitors but these cells also possess limited self-renewal besides ethical issues because of their embryonic/ fetal origin.

Genetic Basis of Inherited Macular Dystrophies and Implications for Stem Cell Therapy

Retinal pigmented epithelial cells and photoreceptors derived from these are some of the most promising cells that may soon be used in the treatment of specific HMD, especially since rapid developments in the field of induced pluripotency have set the stage for the production of patient‐derived stem cells that overcome the ethical and methodological issues surrounding the use of embryonic derivatives.

Generation, Purification and Transplantation of Photoreceptors Derived from Human Induced Pluripotent Stem Cells

Evidence is provided that enriched populations of human photoreceptors can be derived from iPS cells, and it is found that when the FACS purified iPSC derived photoreCEPTs are transplanted into a normal mouse retina and express photoreceptor markers.

Stem cells as a therapeutic tool for the blind: biology and future prospects

This work proves that residual retinal circuits can be reawakened after photoreceptor loss and defines a goal for stem-cell-based therapy to replace photoreceptors in blind patients.

Induction of retinal pigment epithelial cells from monkey iPS cells.

The RPE cells derived from monkey skin with iPS cell technology can be used for autologous or allogeneic transplantation to test the possibility of immune rejection and to evaluate their function in vivo with the same techniques that will be used in clinical trials.

Transplantation of Adult Mouse iPS Cell-Derived Photoreceptor Precursors Restores Retinal Structure and Function in Degenerative Mice

Adult fibroblast-derived iPSCs provide a viable source for the production of retinal precursors and subsequently photoreceptor cells for retinal transplantation to restore retinal function in degenerative hosts.

Long-term Safety and Efficacy of Human-Induced Pluripotent Stem Cell (iPS) Grafts in a Preclinical Model of Retinitis Pigmentosa

This study provides the first direct evidence of functional recovery in a clinically relevant model of retinal degeneration using iPS transplantation and supports the feasibility of autologous iPS cell transplantation for retinal and macular degenerations featuring significant RPE loss.

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

Although extrapolation to humans requires caution, the high transduction efficiency of both rod and cone photoreceptors and the statistically significant reduction of A2E accumulation in the mouse model of STGD1 suggest that lentiviral gene therapy is a potentially efficient tool for treating ABCA4-associated diseases.

Sense and serendipity aid RPE generation.