For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. Now, thanks to the work of researchers at UCLA Broad Stem Cell Research Center, a new lifesaving gene therapy could change all that. The five-year-old was born with ADA-SCID, a … Continue reading How CIRM-funded research brings lifesaving gene therapy to a 5-year-old
Bespoke Gene Therapy Consortium
Bespoke approach to rare diseases
Until recently, “bespoke” meant one thing to me: a custom suit from London’s Savile Row. They’re beautiful—and expensive—so I assumed I’d never own anything bespoke. A tailored approach I was wrong. CIRM is now part of a bespoke effort far more important than clothing. This group aims to create tailor‑made gene therapies for rare diseases. … Continue reading Bespoke approach to rare diseases