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Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease ...
The only cure for painful sickle cell disease today is a bone marrow transplant. On Tuesday, advisers to the Food and Drug Administration will review a gene therapy for the inherited blood ...
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). [ 64 ] The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a ...
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
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.
A sickle allele is always the same mutation of the beta-globin gene (glutamic acid to valine at amino acid six). In contrast, beta-thalassemia alleles can be created by many different mutations including both deletion and non-deletion forms.
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