In this episode, Sarah Blutt explains how this work demonstrates the power of organoids to understand and develop new therapies for diseases.
In this episode, Bikash Pattnaik and colleagues explain that their study encompasses the application of genomic medicine to treat monogenic retinal degeneration using base editing gene therapy delivered through novel nonviral platform.
In this episode, Ushma Neill interviews Elizabeth Jaffee, the Johns Hopkins–based oncologist and immunologist who led the clinical development of a first-gen cancer vaccine for pancreatic cancer and continues to use innovative approaches to identify the complex signaling pathways in tumor cells, the microenvironment, and the immune system toward the generation of cancer immunotherapies. All the while she's been a leader at the local, national, and international level, and currently is the chair of President Biden's Cancer Panel.
In this episode, Ushma Neill interviews the physician scientist and gene therapy, pioneer Dr. Katherine High. After a long career as an academic hematologist, first at the University of North Carolina, then at the University of Pennsylvania and HHMI, Kathy transitioned to a role in industry, first at Spark Therapeutics, then at ASK Biopharma where she led the first FDA approval for gene therapy for a genetic disease.
Neonatologist and pulmonary biologist Jeffrey Whitsett of the Cincinnati Children’s Hospital provided understanding of pulmonary surfactant biochemistry and structural biology that underpins the widespread application and usage of surfactant replacement therapy in neonatal respiratory distress.