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In addition, clinical trials to cure beta thalassemia and sickle cell disease in human patients using CRISPR-Cas9 technology have shown promising results. [ 173 ] [ 174 ] In December 2023, the US Food and Drug Administration (FDA) approved the first cell-based gene therapies for treating sickle cell disease, Casgevy and Lyfgenia .
CRISPR technology is a promising tool not only for genetic disease corrections but also for the prevention of viral and bacterial infections. Utilizing CRISPR–Cas therapies, researchers have targeted viral infections like HSV-1, EBV, HIV-1, HBV, HPV, and HCV, with ongoing clinical trials for an HIV-clearing strategy named EBT-101 ...
Developed from a natural defense mechanism found in bacteria, CRISPR-Cas9 is the most commonly used system, that allows "cutting" of DNA at specific locations and either delete, modify, or insert genetic material. This technology has transformed fields such as genetics, medicine, [179] [180] and agriculture, [181] [182] offering potential ...
CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function ...
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.
Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.
For example, the Berlin Patient has a genetic mutation in the CCR5 gene that suppresses the expression of CCR5. This confers innate resistance to HIV. Modifying human embryos to give the CCR5 Δ32 allele protects them from the disease. An other use would be to cure genetic disorders.
CRISPR gene editing technology in humans has the potential to cause profound social impacts, [112] such as in the long-term prevention of diseases in humans. [113] However, He's human experiments raised ethical concerns the effect are unknown on future generations. [ 112 ]