A scientist at work in a biomedical laboratory. (Photo: Tom Robertson, via Shutterstock)
The California stem cell agency says it is doing “everything” it can to move forward on a gene therapy that has saved the lives of more than 50 persons but which has been pushed aside by the company that has exclusive rights to it. The issue has raised questions about the ethics of withholding care from babies and children suffering from a fatal disease.
Evangelina, a former 'bubble baby,' plays inside a giant plastic bubble. (Photo: Stem Cellar Report, CIRM)
Little Evangelina Padilla-Vaccaro is more than a poster girl for the $12 billion California stem cell agency. She embodies a big bet by the agency that its efforts will conquer at least a few of the terrible diseases that are currently incurable. In the case of Evie, as the eight-year-old is known, she was born with what has come to be described as the bubble baby syndrome, a rare genetic mutation that crippled her immune system to the point that she would have died if left untreated.