Search Results for: dana-farber boston children's
Cerebral adrenoleukodystrophy: How the Cooksons dodged a devastating disease
Heather Cookson believes that if she hadn’t insisted her son Ricky get a brain MRI to investigate his frequent headaches, …Read More
Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits
Cerebral adrenoleukodystrophy (CALD), portrayed in the film Lorenzo’s Oil, is a devastating disorder caused by a mutation on the X …Read More
In a thriving gene therapy program, nursing leadership is the driving force
Gene therapy was made possible by decades of technological advances. But to execute gene therapy at scale? That would not …Read More
Blood donations help Kit manage Diamond-Blackfan anemia — so she can dance, sing, and enjoy life
Every month, Kit Murdoch needs a blood transfusion to stay alive. The 2-year-old has Diamond-Blackfan anemia, a rare genetic blood disorder …Read More
Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history
Ed. Note: This post updates an earlier post from 2018. In a landmark decision today, the Food and Drug Administration …Read More
One-time treatment could block a deadly form of graft-versus-host disease
Even when a bone marrow transplant cures leukemia or lymphoma, patients can still pass away from graft-versus-host disease (GVHD), in …Read More
Playing the long game: An exciting discovery in telomere disease
Each time our cells divide, the protective caps that keep our chromosomes from fraying, called telomeres, lose a bit of …Read More
Walter’s world: ‘Warrior’ toddler doesn’t let a brain tumor stop him
When Walter Merck attempts an obstacle course at his physical therapy appointment, “he just does what he wants,” laughs his …Read More
Tim Springer: Scientist, entrepreneur, and mentor
As an undergraduate in 1966, immunologist, biochemist, and biophysicist Timothy A. Springer, PhD, looked askance at science. The Vietnam War …Read More
New work transforms our knowledge of how blood is formed
The origins of our blood may not be quite what we thought. In groundbreaking research, scientists in the Stem Cell …Read More