Therapy for neuronopathic Gaucher Disease using gene therapy and a specialized neuronal lineage iPS cell that facilitates entry to and engraftment in the brain.
Human Natural Killer cells expressing chimeric antigen receptor that targets aberrant immune system signaling that is involved in auto-antibody production associated with Systemic Lupus Erythematosus.
Quantitatively identifying diffuse white matter anomalies using MRI as a systematic approach provides early identification of the need for developmental deficit interventions in preterm infants.
Perforin inhibition during vaccination can improve vaccine success against a variety of infections and diseases for which sufficiently efficacious immune response has proven difficult
A diagnostic, prognostic, and patient monitoring algorithm that compares text and speech characteristics to a corpus to distinguish patients; validated in suicidal vs. non-suicidal adolescents and epilepsy surgery prediction.
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.