Therapy for neuronopathic Gaucher Disease using gene therapy and a specialized neuronal lineage iPS cell that facilitates entry to and engraftment in the brain.
Perforin inhibition during vaccination can improve vaccine success against a variety of infections and diseases for which sufficiently efficacious immune response has proven difficult
Pharmacologic and genetic inhibition of UBE2N suppresses the function and viability of MDS/AML cells lines and patient samples. A commercially available and a novel compound has been identified.
A newly established link between the mechanistic role of thrombin and fibrinogen and their contribution to AD provides a novel mechanism for oral treatment.