Randall Jeffrey Platt

TL;DR: In vivo as well as ex vivo genome editing using adeno-associated virus, lentivirus, or particle-mediated delivery of guide RNA in neurons, immune cells, and endothelial cells is demonstrated, suggesting that Cas9 mice empower a wide range of biological and disease modeling applications.

TL;DR: In this paper, a Cre-dependent Cas9 knockin mouse was used to study the dynamics of KRAS, p53, and LKB1, the top three significantly mutated genes in lung adenocarcinoma.

TL;DR: In this article, the authors discuss current progress toward developing programmable nuclease-based therapies as well as future prospects and challenges, and discuss the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies.

TL;DR: Current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges are discussed.

TL;DR: The development of light-inducible transcriptional effectors (LITEs), an optogenetic two-hybrid system integrating the customizable TALE DNA-binding domain with the light-sensitive cryptochrome 2 protein and its interacting partner CIB1 from Arabidopsis thaliana, establishes a novel mode of optogenetics control of endogenous cellular processes and enables direct testing of the causal roles of genetic and epigenetic regulation in normal biological processes and disease states.