Immune cells repaired with Crispr to cure diseases - Biotech.com. Thanks to Crispr-Cas9, the DNA cut-and-sew technique, it has been possible to'repair' immune cells by correcting the genetic defects underlying rare diseases.
The technique has been successful both in mice, in which it completely cured the disease, and in human cells taken from affected children and grown in the laboratory. The study highlights that the therapeutic mechanism works in principle, but clinical studies will be necessary to evaluate the safety and duration of the protective effect.