The pharmaceutical industry is abuzz with a latest trend of gene editing, the technology used to modify the human genome. Gene editing has been on the rise since the discovery of the CRISPR-Cas9 system, which is helpful in rewriting genomics using scissors. In the past, scientists have optimized gene therapy to simply treat a disease, however now the scientists are trying to use the technology to cure diseases that were otherwise untreatable.

Recently, a group of scientists were able to use the gene-editing technique to modify the DNA of patients with a rare genetic disorder known as Hunter’s syndrome, and have seen extremely promising results. This rare genetic disorder occurs when the body produces less of a specific enzyme that is necessary for breaking down a waste product. This waste product can build up in the cells, causing symptoms such as developmental delays and joint pain.

The gene-editing treatment has been used to add the missing enzyme back into the patient’s DNA, allowing them to break down the waste product effectively. Since the new DNA is incorporated into the patient’s cells, the benefits of this treatment could remain lifelong.

This new development has shown possible cure for many genetic disorders such as cystic fibrosis, musculodystrophy and sickle cell anemia.

Despite the incredible potential shown by this technology, there are calls from many corners for more scrutinized and stringent regulations around gene editing, to prevent accidental gene mutations while editing. While it is a great achievement in the healthcare sector to have a new and innovative way to correct genetic disorders, it is important to keep in mind the potential risks involved and proceed with the necessary caution.


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The material in this article is written on the basis of another article.

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