Final answer:
In gene therapy strategy for sickile cell disease, c-Myc is typically removed from HbS/HbS iPS cells using gene-editing techniques like CRISPR-Cas9 or RNA interference, although the exact method is not detailed in the provided information.
Step-by-step explanation:
Gene Therapy Strategy for Sickle Cell Disease
In the context of gene therapy strategy for sickle cell disease, the method of ex vivo gene therapy is often utilized. This involves removing somatic cells from the patient, culturing them, and introducing a correct version of the gene into these cells using a vector. Specifically, for induced pluripotent stem cells (iPS cells) containing the mutation for sickle cell disease (HbS/HbS genotype), the oncogene c-Myc needs to be removed or inactivated to avoid cancerous growths when these iPS cells are used in treatments. The detailed mechanism by which c-Myc is removed from the HbS/HbS iPS cells is not provided in the given resources, but would typically involve techniques such as CRISPR-Cas9 gene editing or RNA interference (RNAi), which are standard methods to target and inactivate specific genes within the cells.
The procedure facilitates the insertion of the corrected gene without the risk of uncontrolled cell growth due to c-Myc, and the modified cells are reintroduced into the patient, potentially leading to the production of healthy hemoglobin and alleviation of sickle cell symptoms. This strategic approach aims to provide a long-lasting solution to genetic disorders by enabling the patient's own cells to function correctly.