We report a gene therapy strategy using CRISPR/ Cas9-mediated cellular reprogramming by switching... more We report a gene therapy strategy using CRISPR/ Cas9-mediated cellular reprogramming by switching a mutation-venerable/sensitive cell type to a mutation-insensitive/resistant cell type, therefore restoring tissue architecture and function. We applied this strategy to retinitis pigmentosa (RP), a major cause of blindness characterized by retinal rod photoreceptor degeneration caused by numerous mutations in many genes. By re
We report a gene therapy strategy using CRISPR/ Cas9-mediated cellular reprogramming by switching... more We report a gene therapy strategy using CRISPR/ Cas9-mediated cellular reprogramming by switching a mutation-venerable/sensitive cell type to a mutation-insensitive/resistant cell type, therefore restoring tissue architecture and function. We applied this strategy to retinitis pigmentosa (RP), a major cause of blindness characterized by retinal rod photoreceptor degeneration caused by numerous mutations in many genes. By re
Uploads
Papers by Duc Hoang