Scientific developments in the field of gene editing are opening up prospects for treating genetic diseases, after scientists used advanced technology to edit DNA directly inside the human body.
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In a clinical trial, scientists used "prime editing" technology to target a gene mutation associated with Wilson's disease, a rare disease that leads to the accumulation of copper in the body, particularly in the liver, and can cause damage and neurological complications if left untreated.
The results of this development were published in the journal Nature, amid hopes that the technology will help repair the genetic defect from within the body, rather than extracting cells, modifying them in the laboratory, and then returning them to the patient.
The treatment, developed by the American company Prime Medicine, is based on delivering gene-editing tools to liver cells using fatty nanoparticles.
The "primary editing" technique differs from some traditional gene-editing methods, such as CRISPR-Cas9, in that it allows for specific changes to DNA by replacing, inserting, or deleting parts of it. Therefore, scientists liken it to a word processing program, but at the level of the genetic code.
Reducing the need for complex and expensive laboratory procedures.
Avoid extracting cells, modifying them outside the body, and then re-injecting them.
Reducing the need for some procedures associated with cell therapies, such as chemotherapy used in certain cases to prepare the body to receive the modified cells.
This method may also contribute to reducing the costs of producing gene therapies, thus making them accessible to a larger number of patients in the future.
Despite the hopes associated with the technology, there are still challenges related to the safety and accuracy of the modifications, and the extent to which scientists can deliver the modification tools to the target cells without affecting other parts of the DNA.
"It depends on the type of risk you're willing to accept," said Krishano Saha, a biomedical engineer at the University of Wisconsin-Madison, noting that delivering gene-editing tools precisely to the target organs remains the primary challenge.
Joseph Thom, a biotechnology analyst at TD Cowen, explained that developing gene therapies requires significant investments, but noted that modifying genes directly inside the body could reduce production costs.
Prime Medicine is expected to announce the initial clinical results of the trial next year, coinciding with anticipation of regulatory decisions regarding other CRISPR-based treatments administered directly into the body.
However, proving the long-term efficacy and safety of the treatment is still necessary before determining its ability to provide a sustainable cure for Wilson's disease.
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