The other side of science: CRISPR "genetic scissors" have proven to be hazardous to humans.

The CRISPR technology, often celebrated for its potential to correct genetic defects, faces significant challenges in treating certain genetic disorders. A team of specialists from the ImmuGene program at the University of Zurich recently reported these limitations while studying chronic granulomatous disease (CGD), a rare immune disorder, according to ScienceDaily.

CRISPR's ability to precisely target and edit genetic code allows scientists to replace or restore defective DNA sequences, which is crucial for treating diseases caused by specific mutations. However, in the case of CGD, where a minor DNA defect disrupts the immune response, it was found that CRISPR editing could potentially introduce new and unpredictable genetic changes, limiting its clinical application.

CGD affects approximately 1 in 120,000 people and is caused by the absence of two DNA bases in the NCF1 gene. The missing genetic code disrupts the function of an enzyme complex necessary for fighting infections, making patients vulnerable to life-threatening bacterial and fungal infections. The team, led by Professor Janine Reichenbach, successfully used CRISPR to insert the missing bases into immune cells with the same defect observed in CGD patients, marking a significant breakthrough.

However, some of the corrected cells exhibited new chromosomal abnormalities, where entire segments were missing—a concerning side effect that could have serious health implications, including the potential development of leukemia, as stated in a study published in the journal Communications Biology.

Unintended changes occur due to CRISPR's inability to distinguish the functional NCF1 gene from two nearby pseudogenes. When CRISPR cuts the DNA, it may inadvertently affect these pseudogenes, leading to misplacement of genetic material during the reassembly process. Such mismatches can result in unpredictable and dangerous outcomes, highlighting the need for caution in the clinical use of CRISPR. In response, the team explored alternative methods, including system adjustments and protective elements designed to reduce the likelihood of cuts at multiple gene sites.

Despite these efforts, they found that none of the approaches could completely eliminate the risk of unwanted genetic changes. Co-author Martin Jinek noted that the research underscores both the potential and significant risks associated with CRISPR therapy for CGD and other genetic disorders, adding, "Further technological advancements are necessary to make this method safer and more effective for people in the future."

Gene editing methods like CRISPR remain promising for treating genetic diseases, but this study clearly demonstrates the complexities and risks involved in editing genomic areas containing multiple gene copies or pseudogenes. As research continues, experts are exploring safer solutions in gene editing, such as more precise genome editing tools and improved delivery mechanisms that could potentially bypass some of the existing limitations of the system.

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