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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, 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 ...
CRISPR gene editing is a revolutionary technology that allows for precise, targeted modifications to the DNA of living organisms. 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.
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 ...
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.
Genetic engineering is in widespread use, particularly in agriculture. Human germline engineering has two potential applications: prevent genetic disorders from passing to descendants, and to modify traits such as height that are not disease related. For example, the Berlin Patient has a genetic mutation in the CCR5 gene that suppresses the ...
Fast-paced developments in the CRISPR-Cas9 gene editing technology has increased both the concerns and relevance of this ethical controversy as it has become more popularly used. [ 19 ] [ 20 ] The scientific community recommends continued evaluation of risks and benefits of utilizing genetically modified organisms in everyday life. [ 21 ]
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.