May 9, 2016
(The Scientist) – Traditionally, gene therapy efforts have attempted to treat genetic diseases by modifying DNA inside a patient’s body, but it has been a challenge to deliver the genetic material to all the target tissue, let alone to do so safely. But in recent years, advances in gene editing and stem cell research have enabled scientists to correct genetic defects in a patient’s own cells and grow tissue-specific “organoids” in vitro. These mini organs hold promise for modeling disease, screening drugs, and—potentially—replacing defective tissue in patients.
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