These genetic discoveries
reinforce earlier gene studies implicating abnormal inflammation and fat metabolism in the disease.
Not exact matches
Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the
early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical trial
reinforces the strength of our
gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the
gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»
The special attributes of the ancestral versions of these enzyme superfamlies, and the self -
reinforcing feedback system they would have formed with the first
genes and proteins, would have kick - started
early biology and driven the first life forms toward greater diversity and complexity, the researchers said.
«These
early results in infants
reinforce hope that
gene therapy will prove to be safe and effective at restoring immune function
early in life,» said Brian Sorrentino, M.D., a member of the St. Jude Department of Hematology, who leads this study with Mamcarz.