Long -Term Tests Demonstrate Effective CRISPR Editing Techniques in Treating Thalassemia and Sickle Cell Anemia

 


With the CRISPR technique, human genetics can be edited to remove infectious diseases that are then passed on to offspring. Although newly created in 2012, the impact of CRISPR was so great that its creators Emmanuelle Charpentier and Jennifer Doudna were awarded the 2020 Nobel Prize in Chemistry.


During the ongoing European Hematology Association Congress conference this week, long -term test results showed CRISPR is effective for the long term for treating thalassemia and sickle cell anemia. Two patients were treated in 2019 using CRISPR and to date they are still recovering from this genetic disease.



Next CRISPR was used to treat 44 patients with thalassemia and 31 patients with sickle cell anemia. All 31 sickle cell anemia patients fully recovered after undergoing treatment. Only two thalesemia patients were not successfully treated but their blood transfusion requirements were successfully reduced by up to 89% compared to before. The effectiveness of treatment did not show any decrease 37 months after treatment was received and also without long -term effects.


Phase 3 clinical trials will begin in the United States soon to collect more data to see the effectiveness of the treatment and its impact on patients. CRISPR treatment has been granted swift approval by the FDA with the target that it can be made available to the public as early as 2023.



The use of CRISPR raises various questions about ethics because its long -term effects are still unknown. In 2018, Chinese scientists produced CRISPR babies that are immune to HIV infection. The scientist's announcement was criticized for the risk to the unborn baby. At the same time cancer treatment using CRISPR to treat cancer has already begun. Perhaps in just a few generations more genetic and deadly diseases can be treated as easily as genetic editing alone.

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