The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers at the University of Zurich have now shown that there is a risk of inadvertently introducing other defects.
The CRISPR molecular scissors have the potential to revolutionize the treatment of genetic diseases. This is because they can be used to correct specific defective sections of the genome. Unfortunately, however, there is a catch: under certain conditions, the repair can lead to new genetic defects – as in the case of chronic granulomatous disease. This was reported by a team of basic researchers and physicians from the clinical research program ImmuGene at the University of Zurich (UZH).
Chronic granulomatous disease is a rare hereditary disease that affects about one in 120,000 people. The disease impairs the immune system, making patients susceptible to serious and even life-threatening infections. It is caused by the absence of two letters, called bases, in the DNA sequence of the NCF1 gene. This error results in the inability to produce an enzyme complex that plays an important role in the immune defense against bacteria and molds.
The CRISPR tool works…
The research team has now succeeded in using the CRISPR system to insert the missing letters in the right place. They performed the experiments in cell cultures of immune cells that had the same genetic defect as people with chronic granulomatous disease.
This is a promising result for the use of CRISPR technology to correct the mutation