the world's first CRISPR-based gene therapy: Can genetic defects be treated?

  The UK Medicines Regulatory Agency has approved the world's first CRISPR-based gene therapy. It is specifically designed to treat sickle cell disease and beta-thalassemia, which are genetic disorders that affect red blood cells.




Hemoglobin is found in red blood cells and carries oxygen throughout the body. Errors in the hemoglobin gene lead to the formation of fragile red blood cells, leading to a lack of oxygen in the body, a condition known as anemia.


Patients with sickle cell disease also suffer from inflammation and severe pain because the sickle cells form clots that impede blood flow, and patients with sickle cell beta thalassemia are forced to undergo blood transfusions every three to four weeks.


CRISPR is a gene-editing technology that can modify the DNA of living organisms, including humans, and scientists are trying to use it to treat diseases, although there are concerns.

CRISPR is cheap, easy to use, and allows scientists to edit genes using “scissors.” CRISPR can detect genetic abnormalities and replace them with other elements of DNA.


CRISPR technology relies on an enzyme called Cas9, which uses a guide RNA molecule to target the desired section of DNA and edit it to delete the gene or create the desired sequence.


CRISPR technology allows scientists to edit the DNA of cells with unprecedented precision. Scientists use CRISPR technology to correct genetic problems in human embryos and thus treat diseases.


This technology will open new horizons in genomic medicine, as scientists will actually be able to alter and modify target genes quickly and efficiently.


However, there are concerns about using this technology in human germ cells and early-stage embryos, where changes could be passed on to the next generation and used to produce so-called "personalized babies."


It is necessary to ensure the safety of CRISPR. To be sure, gene editing is effective and does not lead to changes in other parts of the genome that could cause harm.


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