CTX۰۰۱ is CRISPR/Cas۹-based treatment to Increasedfetal hemoglobin for Treatment of sickle cell anemia

Sickle cell disease (SCD) is an inherited severe monogenic disease that iscaused by a point mutation in the β-globin chain. This mutation replaces thesixth amino acid in the beta chain with the valine and eventually causes thesickle shape of sickle hemoglobin (HbS or α۲βˢ۲) when it loses its oxygen.Currently available treatments for SCD include medications that do notaddress the underlying cause of the disease. It is also difficult to transplant anallogeneic bone marrow, which is the only possible treatment for SCD, as it isdifficult to find a donor. However, using the CTX۰۰۱ We can use the patient'scells. According to the researchers, CD۳۴+ cells (a type of undifferentiatedmultipotent hematopoietic stem cells) are collected from the blood of a personwith SCD and then CRISPR/Cas۹ delivered via electroporation. CRISPR/Cas۹edited stem cells cryopreserved and stored until infusion. CTX۰۰۱ is thawedand infused into the patient after receiving high-dose myeloablative therapy.CRISPR/Cas۹ genome editing technology that enables rapid and increasinglyaccurate manipulation of the genome can increase the level of fetalhemoglobin (HbF or α۲γ۲) via the NHEJ mechanism knocking down/silencingof BCL۱۱A gene, which is one of the most potent repressors of HbF. Anincrease in HbF in patients with SCD causes showing fever complications.CTX۰۰۱ (that is an autologous CRISPR/Cas۹ gene-edited hematopoietic stemcell therapy, which is a developing treatment method for patients sufferingfrom β-thalassemia and SCD), uses the CRISPR-Cas۹ gene-editing tool tomodify a patient's hematopoietic stem cells to produce high levels of HbF inred blood cells.

کلیدواژه ها:

SCD ، HbF ، CRISPR/Cas۹ ، CTX۰۰۱ ، Hematopoietic stem cell