Many disorders and life-threatening diseases could be cured by replacing or fixing dysfunctional cells. We aim to uncover novel ways to provide new tissues and cells to the body while minimizing organ rejection.

Gene-editing technology partially restores visionin blind animals

Along with Professor Juan Carlos Izpisua Belmonte, first authors Jun Wu, Reyna Hernandez-Benitez, Keiichiro Suzuki and colleagues discovered the ability to, for the first time, insert DNA at a target location into the nondividing cells that make up the majority of adult organs and tissues. The technique, which the team showed was able to partially restore visual responses in blind rodents, will open new avenues for basic research and a variety of treatments, such as for retinal, heart and neurological diseases.

Until now, techniques that modify DNA—such as the CRISPR-Cas9 system—have been most effective in dividing cells, such as those in skin or the gut, using the cells’ normal copying mechanisms. The new Salk technology, which they named HITI, is 10 times more efficient than other methods at incorporating new DNA into cultures of dividing cells, making it a promising tool for both research and medicine. More importantly, HITI represents the first time scientists have managed to insert a new gene into a precise DNA location in adult cells that no longer divide, offering new possibilities for therapeutic applications in these cells.

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