Mark K. Schlegel

Prevention of prion disease symptoms in mice with deep PrP lowering

This study demonstrates that deep PrP lowering with a potent siRNA prevents prion disease symptoms in mice and extends survival in symptomatic animals.

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene

This paper introduces a method to increase the proportion of correctly repaired hepatocytes in the liver through transient gene inhibition and transgene delivery, achieving a 25% increase in targeted cells and five-fold transgene expression.

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

This paper develops an RNAi-based rheostat to dynamically control AAV-delivered transgene expression using siRNA or shRNA and REVERSIR technology for induction.