In her home on the island of Kauai with her husband Chris Constantino at her side and her two children playing nearby, Nichelle Obar breaks the illusion that nothing was ever amiss in her family's slice of paradise. "It all happened so fast," she says, explaining the cascade of news and life-and-death decisions she and … Continue reading Looking Back in Gratitude: One Family, One Trial, and Moving Science Forward
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 measure, a devastating disease. Caused by four missing or mutated genes, it almost always leads to death before or shortly after birth. Treatment … Continue reading Surviving with Joy
Dr. Tippi Mackenzie, UCSFImage Credit: UCSF Each year, around 24,000 women in the US lose a pregnancy. One reason for this unfortunate occurrence are metabolic disorders, one of which is known as Sly syndrome and is caused by a single genetic mutation. In Sly syndrome, the body's cells lack an enzyme necessary for proper cell … Continue reading Stem cell transplant in utero offers potential treatment for congenital diseases
CIRM's 2018 Annual Report sums up the agency accomplishments over the last year in just 24 pages, The report look at the highlights, progress, and the impact CIRM has had on stem cell research over the last year. But it’s also a look forward at the challenges ahead and the people who will help us … Continue reading CIRM’s 2018 Annual Report
There were lots of CIRM mentions in the news this week. Here are two brief recaps written by Karen Ring to get you up to speed. A third story by Todd Dubnicoff summarizes an promising finding related to heart disease by researchers in Singapore. CIRM-funded “bubble baby” disease therapy gets special designation by UK. … Continue reading Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target