Journal of Cell Science & Therapy
Open Access

Gene-Editing Approaches in Cell Therapy: Transforming the Treatment of Blood Disorders

Julie Teerada^{* *}Correspondence: Julie Teerada, Department of Tumor Biology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany, Email:

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Description

Gene editing is revolutionizing the field of cell therapy, especially in the treatment of blood disorders. Diseases like sickle cell anemia, beta-thalassemia, and certain types of leukemia are caused by genetic mutations that disrupt normal blood cell function. Traditional treatments, such as blood transfusions or bone marrow transplants, have limitations, and there is a need for therapies that address the root cause of these disorders.

Gene-editing technologies

CRISPR-Cas9 is the most widely known gene-editing tool, offering precise modifications of specific genes by targeting a specific sequence of DNA. The system works by using a guide RNA to direct the Cas9 enzyme to the target gene, where it induces a double-strand break. The cell then repairs this break, often introducing a mutation or correction. In cell therapy, CRISPR-Cas9 is used to edit Hematopoietic Stem Cells (HSCs), the progenitors of all blood cells. For diseases like sickle cell anemia and beta-thalassemia, the mutation in the hemoglobin gene can be corrected in HSCs ex vivo, after which the modified cells are transplanted back into the patient, providing long-term correction of the disease.

Unlike CRISPR-Cas9, which creates double-strand breaks, base editing directly converts one DNA base pair into another without causing DNA breaks. This is particularly useful for point mutations, which are the cause of many blood disorders. Base editing provides higher accuracy and fewer unintended genetic changes, making it a potential tool for therapies aimed at correcting specific mutations in blood disorders. Prime editing is a newer and even more precise gene-editing technique. It allows for the correction of a broader range of mutations with fewer offtarget effects than CRISPR-Cas9 or base editing. Prime editing uses a "prime editor" that can directly rewrite the target DNA sequence. This tool holds immense potential for fixing a variety of mutations, including those that cause blood disorders.

Applications

Sickle cell anemia is caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and misshaped red blood cells. Traditional treatments like blood transfusions and bone marrow transplants are not curative and often come with complications. However, using CRISPR-Cas9 to correct the sickle cell mutation in patient-derived stem cells has shown potential in clinical trials. Researchers are also exploring gene therapy approaches, such as reactivating fetal haemoglobin production to compensate for the sickle hemoglobin.

Beta-thalassemia is another blood disorder caused by mutations in the beta-globin gene. Gene-editing techniques, particularly CRISPR-Cas9, are being tested to insert functional copies of the beta-globin gene or to fix the mutations in the patient’s own hematopoietic stem cells. Early results have been encouraging, with some patients achieving transfusion independence after receiving gene-edited cells. In addition to inherited blood disorders, gene editing is also being used to target hematologic cancers such as leukemia and lymphoma. One approach involves editing immune cells (like T-cells) to better recognize and attack cancer cells. Another strategy involves knocking out genes that inhibit immune responses or adding genes that enhance the immune system's ability to fight tumors.

Conclusion

Gene-editing technologies are reshaping the landscape of blood disorder treatment, providing potential cures by directly correcting the genetic defects at the heart of these diseases. Advances in CRISPR-Cas9, base editing, and prime editing are paving the way for more effective, personalized treatments. While challenges remain, the progress made so far suggests a future where genetic blood disorders can be treated at their genetic root, providing hope for patients who currently rely on lifelong management strategies.