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Sickle cell disease (SCD), also simply called sickle cell, is a group of hemoglobin-related blood disorders that are typically inherited. [2] The most common type is known as sickle cell anemia. [2] Sickle cell anemia results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2]
Clinical trials for one of the first attempts in somatic cell genome editing using CRISPR technology are for the treatment of sickle cell disease. he and his colleagues have published multiple peer-reviewed studies identifying gaps in knowledge of trial participants that could prevent them from giving adequately informed consent.
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.
A study published in 2021 found that 50% of sickle cell patients reported having to wait at least two hours before their pain was treated, despite medical guidelines recommending such patients in ...
In 2021, the US Food and Drug Administration approved a clinical trial for an experimental CRISPR-based therapy for sickle cell disease developed by a consortium including the IGI, UCSF Benioff Children's Hospital, and the UCLA Broad Stem Cell Research Center. [48]
When combined with Hemoglobin S (β^6Glu → Val) it causes a severe form of Sickle cell disease known as Hemoglobin S/O-Arab. Detection of Hb O-Arab can be carried out with a blood test, identifying the carries of hemoglobinopathies, so as to inform patients their chances of producing an affected child and ensure appropriate guidance is given.
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