CRISPR used to partially restore vision in blind rats

Suzuki, K., et. al. Nature (2016) https://www.ncbi.nlm.nih.gov/pubmed/27851729

In vivo integration of a transgene has historically been the main challenge to advancing gene therapy due to its inefficiency, especially in non-dividing cells.  Suzuki et. al. developed a CRISPR/Cas9 system using a novel homology-independent targeted integration strategy to knock-in DNA sequences in both dividing and non-dividing cells.  In order to demonstrate the techniques therapeutic potential, Suzuki et. al. used this technique to partially restore retinal function in a retinal degeneration rat model.

Author: Advanced Analytical

Advanced Analytical Technologies, Inc. (AATI) simplifies complex genomics workflows to accelerate research and discovery in pharmaceuticals, life science, biofuels, biotechnology and healthcare.

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