Small RNAs aid cell transplants

摘要

With the help of small RNA molecules called micro RNAs, a metabolic and neurodegenerative disorder might be treated using gene therapy.rnPatients with Krabbe's disease lack a functioning version of an enzyme called GALC. Transplants ofrngenetically engineered blood stem cells that later develop into white blood cells could potentially deliver a corrected version of GALC. But expressing the protein in the stem cells kills them.