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In 2023, the first drug making use of CRISPR gene editing, Casgevy, was approved for use in the United Kingdom, to cure sickle-cell disease and beta thalassemia. [13] [14] Casgevy was approved for use in the United States on December 8, 2023, by the Food and Drug Administration. [15]
Using CRISPR, it edits the DNA found in a patient’s stem cells to remove the gene that causes the disease. “The patient is their own donor,” Thompson said.
Gene editing is a potential approach to alter the human genome to treat genetic diseases, [40] viral diseases, [41] and cancer. [42] [43] As of 2020 these approaches are being studied in clinical trials. [44] [45]
The new exa-cel treatment under FDA consideration can use the patient’s own stem cells. Doctors would alter them with CRISPR to fix the genetic problems that cause sickle cell, and then the ...
Modifying human embryos to give the CCR5 Δ32 allele protects them from the disease. An other use would be to cure genetic disorders. In the first study published regarding human germline engineering, the researchers attempted to edit the HBB gene which codes for the human β-globin protein. HBB mutations produce β-thalassaemia, which can be ...
The “New York patient.”The first woman and person of mixed-race ancestry possibly to be cured, she was diagnosed with leukemia in 2017 and received a stem cell transplant augmented with ...
With the discovery of various types of immune-related disorders, there is a need for diversification in prevention and treatment. Developments in the field of gene therapy are being studied to be included in the scope of this treatment, but of course more research is needed to increase the positive results and minimize the negative effects of gene therapy applications. [27]
However, the therapy exceeded all expectations and at the end of July 2019, Gray was announced as the first patient to be treated for sickle-cell disease using the CRISPR-Cas9 gene-editing technology. [2] Thanks to her gene-edited cells, Gray has been cured of the disease and now lives a symptom-free life.
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