Prenatal Gene Therapy for Sickle Cell Disease with Dr. Panicos Shangaris King's College London TRANSCRIPT

Dr. Panicos Shangaris is from King's College London, where he is a senior clinical lecturer and consultant in maternal and fetal medicine.  He discusses the treatment options for sickle cell disease, an inherited blood condition, and his work on the Pericles project, which aims to treat it before birth. In-utero gene therapy offers a third option for parents of an affected fetus alongside postnatal treatment or pregnancy termination. Panicos emphasizes key advantages of fetal therapy within the first 16 weeks of gestation, the need to address ethical concerns, and ensuring the safety of both the mother and fetus during the procedure. 

Panicos explains, "So sickle cell disease is an inherited condition that affects the blood and the molecule in the red cell, which is basically the red blood cell that carries oxygen. So what happens is the red cell has a biconcave shape, and for people who have sickle cell disease, that biconcave shape is lost, and the erythrocyte red cells are sickle-shaped. So because of that, the sickle-shaped red cells have difficulty going through small capillaries and small vessels. They can cause strokes, they can cause pain because they block the blood flow to the small capillaries."

"Yes, so the cause is because of an abnormal gene, which basically makes the hemoglobin, the molecule that carries oxygen around the different organs and around the body, instead of there being a problem in the genetic code, if I can put it like that. And that's why the erythrocyte, the red cells, have an abnormal shape. Very well-known what's causing it: a genetic problem."

"So Pericles stands for prenatal therapy for Sickle Cell Disease. And what we are trying to achieve is to offer a third option to people who might carry a fetus affected by sickle cell disease. At the moment, the two options are stopping the pregnancy, termination of pregnancy, or having an affected fetus and an unaffected baby and sick postnatal care. So our aim is to offer a third option, which is correcting the disease inside the womb so that the parents have a healthy baby. So correct the gene while the baby is inside the womb. And this is the main aim of the project."

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