DNA mutations can be corrected very accurately with the CRISPR gene scissors, thus rectifying the causes of genetic diseases. However, a team of researchers and physicians from the ImmuGene clinical research program at the University of Zurich (UZH) has now discovered during laboratory tests that serious new genetic defects may arise unintentionally.
According to a report from the UZH, the discovery was made during correction of the DNA sequence of the NCF1 gene. This sequence is responsible for the rare hereditary condition chronic granulomatous disease. Team leader Janine Reichenbach describes the successful repair as “a promising result”. She is a professor of somatic gene therapy at the University Children’s Hospital Zurich and the Institute for Regenerative Medicine at UZH.
However, the repaired cells displayed defects afterward. This is due to a specific characteristic of the NCF1 gene: it appears in the chromosome three times, once as an active gene and twice in the form of pseudogenes.
Since the CRISPR gene scissors cannot differentiate between these, it occasionally cuts the DNA strand in several locations. When reassembled, entire gene sections are misaligned or missing. This can lead to unpredictable medical consequences. Various measures have been attempted to mitigate this risk without success thus far.
Co-author Martin Jinek, a professor at the UZH Department of Biochemistry, commented: “This study highlights both the promising and challenging aspects of CRISPR-based therapies.” He added: “However, further technological advances are needed to make the method safer and more effective in the future.” ce/mm
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