Dr. Tippi MacKenzie (left) of UCSF Benioff Children’s Hospital San Francisco, visits with newborn Elianna and parents Nichelle Obar and Chris Constantino. Photo by Noah Berger Alpha thalassemia major is, by any stretch of the imagination, a dreadful, heart breaker of a disease. It's caused by four missing or mutated genes and it almost always … Continue reading Surviving with Joy
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Promising start to CIRM-funded trial for life-threatening blood disorder
Aristotle At CIRM we are always happy to highlight success stories, particularly when they involve research we are funding. But we are also mindful of the need not to overstate a finding. To quote the Greek philosopher Aristotle (who doesn’t often make an appearance on this blog), “one swallow does not a summer make”. In … Continue reading Promising start to CIRM-funded trial for life-threatening blood disorder
Here’s a new gene editing strategy to treat genetic blood disorders
If you’re taking a road trip across the country, you have a starting point and an ending point. How you go from point A to point B could be one of a million different routes, but the ultimate outcome is the same: reaching your final destination. Yesterday scientists from St. Jude Children’s Research Hospital published … Continue reading Here’s a new gene editing strategy to treat genetic blood disorders
Potential treatment for sickle cell disease close
Scientists at UCLA found a potentially treatment for sickle cell disease, a new method of gene therapy that can correct the genetic mutation and help the body on its way to generate normal, healthy blood cells for the rest of the patient’s life. The study, funded in part by CIRM and reported in the journal … Continue reading Potential treatment for sickle cell disease close