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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 .
The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its ...
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 ...
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]
Genetic human enhancement emerges as a potential frontier in disease prevention by precisely targeting genetic predispositions to various illnesses. Through techniques like CRISPR, specific genes associated with diseases can be edited or modified, offering the prospect of reducing the hereditary risk of conditions such as cancer, cardiovascular ...
Studies with monkeys show that injecting high-insulin-producing forms of these cells into the animals can “cure” type-1 diabetes for about six months. Human trials are underway.
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
Cure Rare Disease is a non-profit biotechnology company based in Boston, Massachusetts that is working to create novel therapeutics using gene therapy, gene editing (CRISPR technology) and antisense oligonucleotides to treat people impacted by rare and ultra-rare genetic neuromuscular conditions.