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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 .
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 ...
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]
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.
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 ...
Engineered gene drives using CRISPR-cas9 are currently being tested and have been proposed as strategies to eliminate invasive species and disease vectors. By genetically modifying an organism to express an endogenous sequence-specific endonuclease, a target (such as a fertility gene) can be cleaved on the opposite chromosome. [64]
The German man’s donor had just one copy of the CCR5 gene, meaning his immune cells most likely have about half the normal quantity of that protein. In addition, he had only one copy of the gene ...
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.