The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease.
University of ZurichNov 6 2024 However, researchers at the University of Zurich have now shown that there is a risk of inadvertently introducing other defects.
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.
This is a promising result for the use of CRISPR technology to correct the mutation underlying this disease." … but unfortunately, it's not perfect Interestingly however, some of the repaired cells now showed new defects. Entire sections of the chromosome where the repair had taken place were missing. The reason for this is the special genetic constellation of the NCF1 gene: it is present three times on the same chromosome, once as an active gene and twice in the form of pseudogenes. These have the same sequence as the defective NCF1 and are not normally used to form the enzyme complex.
Chromosome Chronic Chronic Granulomatous Disease CRISPR DNA Enzyme Gene Genome Research Technology
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