Gene Therapy in Thalassemia (Zynteglo®) थैलेसीमिया में जीन थेरेपी تھیلیسیمیا میں جین تھراپی

#Zynteglo is a gene therapy developed by Bluebird Bio. It's designed to treat transfusion-dependent beta-thalassemia, a genetic blood disorder. Zynteglo works by introducing a functional copy of a specific gene into a patient's #stemcells, aiming to produce functional hemoglobin and reduce the need for blood transfusions. It's one of the most expensive drugs globally, with an initial price set at around #2millionUSD per treatment. Prices for pharmaceuticals can change over time due to various factors such as negotiations, healthcare policies, or changes in market conditions. It involves a process called ex vivo gene therapy. It works by modifying the patient's own hematopoietic stem cells outside the body (ex vivo). First, a patient's stem cells are collected from their bone marrow. Then, using a viral vector, the cells are genetically modified in a laboratory to introduce a functional copy of the gene responsible for beta-thalassemia. Finally, these modified cells are infused back into the patient's bloodstream, where they are expected to produce normal hemoglobin. Stem cell harvesting for Zynteglo involves a process called #apheresis, where hematopoietic stem cells (#HSCs) are collected from the patient's blood. The patient first undergoes a procedure to stimulate the production of stem cells by #GCSF and #Plerixafor in the bone marrow. Then, during apheresis, blood is withdrawn from the patient through a vein, passed through a machine that separates out the stem cells, and the remaining blood is returned to the patient. In the case of Zynteglo, a process called "#nonmyeloablative #conditioning" is used. This conditioning involves preparing the patient's bone marrow for the infusion of the genetically modified stem cells without completely destroying their existing bone marrow cells. It's a milder form of conditioning compared to traditional myeloablative conditioning used in some other stem cell transplant procedures. Non-myeloablative conditioning aims to create space within the bone marrow for the newly modified stem cells to engraft and grow without completely wiping out the patient's existing stem cells. Zynteglo does not use #CRISPR-Cas9 technology. Instead, it employs a different method involving a viral vector to introduce a functional copy of the gene responsible for beta-thalassemia into the patient's hematopoietic stem cells. Common #side_effects reported in clinical trials included headache, bone pain, abdominal pain, joint pain, and fever. Additionally, there can be risks associated with the procedure itself, such as potential complications from the stem cell collection or infusion process.