Theresa A. Storm

TL;DR: The risk of oversaturating endogenous small RNA pathways can be minimized by optimizing shRNA dose and sequence, as exemplified here by the report of persistent and therapeutic RNAi against human hepatitis B virus in vivo.

TL;DR: All human solid tumor cells require CD47 expression to suppress phagocytic innate immune surveillance and elimination, showing that CD47 is a commonly expressed molecule on all cancers, its function to blockphagocytosis is known, and blockade of its function leads to tumor cell phagcytosis and elimination.

TL;DR: RAAV9, as well as rAAV8, is a robust vector for gene therapy applications and rAAVs9 is superior to rAAv8 specifically for cardiac gene delivery by systemic vector administration.

TL;DR: This study validates DNA family shuffling and viral peptide display as two powerful and compatible approaches to the molecular evolution of novel AAV vectors for human gene therapy applications.

TL;DR: This small fraction of integrated genomes greatly decreases the potential risk of vector-related insertional mutagenesis associated with all integrating vectors but also raises uncertainties as to whether rAAV-mediated hepatic gene expression can persist lifelong after a single vector administration.