 The most challenging thing with sickle cell is understanding that, you know, some days I'm going to have up days and some days I'm going to have down. This is your body. This is what you use every day. Having sickle cell, you're not allowed to... I feel like enjoy some of the little things that other people enjoy. I feel like obviously we've been dealt a crappy hand of cards and, you know, the goal is to play to the best capabilities that you can. Sickle cell anemia is a terrible disorder. It is a hereditary disorder. That is, it's something that occurs in one's gene inherited from parents. Yeah, worldwide it's the most common hereditary disease, so many millions worldwide. In Africa alone, 250,000 newborns are affected annually. So the homework of sickle cell disease is pain. It happens when after the hemoglobin molecule, which is specifically designed to bring oxygen to our vital organs, delivers the oxygen, the sickle mutation, which is the change in the DNA, creates this long strand, making the round, flexible red blood cell into a very sticky, stiff, sickle-shaped, crescent-shaped red blood cell, and that blocks blood flow. So when you don't get enough oxygen to a tissue, causes pain. So patients who have sickle cell disease experience pain, they experience damage to vital organs where blood flow can be altered by the sickle red blood cells. Lung problems, when you don't get enough blood to your brain, causes a stroke. These are all common complications that affect the quality of life, not just the lifespan in a person with sickle cell disease. I feel like I've experienced some really crazy amounts of pain. I've had both my lower extremities, they had infarctions in them, so imagine both your legs just being broken at the same time. It was the worst pain you can think of. And then when my chest, my pericardous, I could barely breathe. I was in constant pain all the time, so there were some days I just felt like a zombie. We know that if you correct that mutation, you take away the disease. So anyone could imagine that if it was possible to correct the gene in one's own cells, that you should be able to cure the disease. So when we first heard about CRISPR-Cas9, we got very excited because that could target the sickle gene, correct the problem, and then restore healthy red blood cells in the circulation. There are three groups of scientists and clinical investigators working together to accelerate the development of this technology in humans with sickle cell disease. So one group at UCLA will be involved with manufacturing that is producing the blood stem cells after correcting the sickle gene, and they are expert in that activity. The group here in Oakland are experts in hematology and sickle cell disease, and ultimately will lead a clinical trial when we begin to treat patients. And then the anchor, I would say, is the IGI, the Innovative Genomics Institute at UC Berkeley, that really brought that technology to us, and they are leading the effort in documenting at the DNA level how many of these blood stem cells we've corrected. We don't have enough therapies for sickle cell disease. If it's related to societal pressures in underprivileged, underrepresented populations in the United States, how rewarding would be to deliver this amazing cure to this underserved population. It's justice, right? It's the first hereditary disease ever defined in human beings primarily affects African Americans and underserved population, has difficult access to healthcare in some cases. Now finally we have a cure that we can offer the majority of patients, at least in our country. I mean that would be terrific.