Results for Gene and Small Molecule Based Medicines

In this news roundup, we celebrate research tools new and old, from RNA sequencing to gene editing to the best tool of all: collaboration.

Learn about novel findings and new funding from our Children’s Hospital of Philadelphia researchers.

Meet our featured Faculty Spotlight scientist for June, Will Bailis, PhD.

Researchers eager to understand circRNAs’ unique set of properties and their functional roles.

Learn about developments in chromatin rebuilding, novel hemophilia treatment, linking heart and kidney disease, and genetic sequencing.

New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.

Synapses were firing throughout the conference room in the Colket Translational Research Building as attendees at the 2017 Research Institute Scientific Symposium held May 2 learned about their colleagues' intriguing research endeavors. The four sessions' themes aligned smartly with the Research Institute's strategic planning process and overall mission of the Children's Hospital of Philadelphia.

The list of genes affecting blood pressure is expanding, as researchers pursue likely targets for therapeutics already in existence or in development.