27-Jan-2021 | Market Research Store

Researchers are on the run find a perfect treatment for severe red blood congenital diseases such as sickle cell anemia or Thalassemia. Recently, a team from theUniversity of Illinois Chicago has found the modification of the DNA of blood cells from the patients to help develop an improved sickle cell disease treatment.According to Dr. Damiano Rondelli, the latest study shows the role of CRISPR and CRISPR-associated genes (Cas) technology —CRISPR-Cas9 technique— in editing the genes in the blood of beta thalassemia and sickle cell disease patients to help them cure.

The team has already received a Nobel Prize in Chemistry in 2020 for their new finding. The CRISPR-Cas9 Gene Editing was used by the researchers in deleting the gene BCL11A for modifying the DNA of stem cells in Sickle Cell Disease and beta-Thalassemia patients. The gene BCL11A was majorly responsible forsuppressing fetal hemoglobin production. The modification helped the stem cells produce fetal hemoglobin such that the effect of defective hemoglobin could be overcome. The advantage of the latest approach is that it uses the patient’s cells instead of relying on a donor and there is no need for a viral vector like the other gene therapies as well. Additionally, the electroporation process helps lower the risk of off-target gene activation.

The UIC hopes that they will have the study expanded across the globe to become a commercially viable treatment. This new procedure is anticipated to become a part of cellular therapy owing to the success experienced in stem cell transplantation. In the sickle cell patients, transplant is the only cure but finding a suitable donor is a huge challenge. Thus, lately 50% compatible donors also termedas haploidentical donors are used. However, there are endless barriers that can inhibit acceptance of a donor-derived transplant. Thus, the researchers hope that the latest gene-editing procedure can become a game changer for the world health.