Surviving with Joy

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

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

One-Time, Lasting Treatment for Sickle Cell Disease May be on Horizon, According to New CIRM-Funded Study

For the nearly 1,000 babies born each year in the United States with sickle cell disease, a painful and arduous road awaits them. The only cure is to find a bone marrow donor—an exceedingly rare proposition. Instead, the standard treatment for this inherited blood disorder is regular blood transfusions, with repeated hospitalizations to deal with … Continue reading One-Time, Lasting Treatment for Sickle Cell Disease May be on Horizon, According to New CIRM-Funded Study