Researchers describe how a new «universal» strategy for CRISPR - Cas9 -
mediated gene correction in patient derived stem cells may represent a promising therapeutic option
Not exact matches
UW Medicine researchers David Mack, Melissa Goddard, Jessica Snyder, Matthew Elverman, and Valerie Kelly co-authored the report, «Systemic AAV8 -
mediated gene therapy drives whole - body
correction of myotubular myopathy in dogs.»
The degree of phenotypic
correction of murine ß — thalassemia intermedia following lentiviral -
mediated transfer of a human γ — globin
gene is influenced by chromosomal position effects and vector copy number.