genetic disease

by Stanford University Medical Center edited by Sadie Harley, reviewed by Andrew Zinin Credit: Pixabay/CC0 Public Domain An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a Phase I clinical trial has shown. While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease […]

by Stanford University Medical Center edited by Sadie Harley, reviewed by Andrew Zinin An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a Phase I clinical trial has shown. While the researchers tested the protocol on patients with Fanconi anemia, a genetic disease that makes standard stem

by Miriam Fauzia edited by Sadie Harley, reviewed by Andrew Zinin DNA, which has a double-helix structure, can have many genetic mutations and variations. Credit: NIH Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency—a promising leap toward treating humans with this rare inherited disease.