Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration.

@article{Wiley2016UsingPI,
  title={Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration.},
  author={Luke A Wiley and Erin Rae Burnight and Arlene V. Drack and Bailey B Banach and Dalyz Ochoa and Cathryn M. Cranston and Robert A Madumba and Jade S East and Robert F. Mullins and Edwin M. Stone and Budd A Tucker},
  journal={Human gene therapy},
  year={2016},
  volume={27 10},
  pages={835-846}
}
Juvenile neuronal ceroid lipofuscinosis (JNCL) is a childhood neurodegenerative disease with early-onset, severe central vision loss. Affected children develop seizures and CNS degeneration accompanied by severe motor and cognitive deficits. There is no cure for JNCL, and patients usually die during the second or third decade of life. In this study, independent lines of induced pluripotent stem cells (iPSCs) were generated from two patients with molecularly confirmed mutations in CLN3, the gene… CONTINUE READING
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