Gene editing in hematopoietic stem cells
Learn about recent breakthroughs in treating blood disorders including sickle cell disease and immune deficiencies with CRISPR-Cas9 technology.
Zulema Romero Garcia, PhD, presents her work focused on gene editing and cell therapy, particularly for treating genetic diseases such as sickle cell disease and CD3δ severe combined immunodeficiency (SCID). She discusses the use of CRISPR/Cas9 technology to edit hematopoietic stem cells, aiming to correct genetic mutations and improve patient outcomes. For sickle cell disease, the approach involves editing the BCL11A gene to induce fetal hemoglobin production, which prevents the sickling of red blood cells.
Dr. Romero also highlights recent advancements in base editing and prime editing, which avoid double-strand breaks and reduce toxicity. These techniques are being used to correct specific mutations, such as those causing SCID. Experimental results demonstrate successful engraftment and gene correction in mouse models, with high levels of gene editing maintained post-transplantation. Additionally, the restoration of T cell development and function in edited cells was observed, indicating the potential for effective gene therapy. Dr. Romero emphasizes the importance of optimizing gene editing techniques and scaling up for clinical applications while ensuring safety and efficacy through rigorous testing.
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