What are the latest, modern and effective treatments for treating thalassemia and sickle cell anemia? If you find new research, please send it to me
Hello Rabab,
There are a huge of research and publications regarding the hemoglobinopathies for treatment and diagnosis. Recently, the American FDA approved one new and effective gene therapy medication for the Sickler patients by using CRISPR-CAS 19 technique. The researcher develops and use this a mazing technique to treat the defective gene in these diseases. Simply, you can search in the Google Scholar for any publication you need, and this is just a link for the latest treatment.
"Novel curative treatments to cure patients with sickle cell disease and transfusion dependent thalassemia".
Good Luck.
The latest advancements in the treatment of thalassemia and sickle cell anemia have seen significant progress, particularly with the advent of gene therapy and CRISPR technology.
Hello,
A clinical trial using CTX001 to treat beta thalassemia is currently underway, co-sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. This trial is intended to assess the safety and efficacy of this method, and the results will illuminate options for future therapeutic applications of CTX001. The trial has shown promising results in beta thalassemia patients, one year after their treatment, including increased levels of hemoglobin and transfusion-independence.
CTX001 therapy involves the extraction of a patient’s hematopoietic stem cells, execution of the CRISPR-Cas9-directed modification, and reintroduction of the edited cells into the patient’s body. CTX001 has been successfully used in transgenic mice to correct sickle cell disease, with clinically relevant efficiency.
In Iraq, currently, patients with sickle cell anemia and thalassemia are treated by stem cell transplantation, noting that the procedure is financially expensive and takes place in specialized centers outside Iraq, such as Turkey, for example.