Molecular Medicine and Gene Therapy

Brad A. Amendt, PhD

  • microRNA gene therapies for tissue engineering
  • Development of 3D printed scaffolds and gene therapies for cleft palate repair
  • Bone and tissue regeneration using new microRNA biotechnologies

Botand Banfi, MD, PhD

  • Molecular mechanisms that underlying defects in cystic fibrosis and deafness, with a focus on ROS and NADPH oxidase

Adam Dupuy, PhD

  • Use of sleeping beauty transposon system to generate tissue specific-models of cancer in mice

John F. Engelhardt, PhD

  • Development of gene therapies for inherited and environmentally induced diseases
  • Development of recombinant adeno-associated viral vectors for gene therapy
  • Investigation of the pathogenesis and treatment of cystic fibrosis lung disease
  • Mechanisms underlying ischemia/reperfusion injury and applications of gene therapy in the liver

C. Andrew Frank, PhD

  • Use of Drosophila genetics and pharmacology to understand signaling pathways that link synaptic instability to disorders of the nervous system

Tom Rutkowski, PhD

  • Mechanistic links between endoplasmic reticulum homeostasis and lipid metabolism in the liver
  • Mechanisms whereby dysregulation of the ER stress response contributes to diseases of liver function (metabolic syndrome, hepatitis, alcohol steatosis)

Ling Yang, PhD

  • . Cellular and molecular mechanisms of Nonalcoholic fatty liver disease (NAFLD);
  • . Organelle function in the context of obesity and diabetes;
  • . The role of organelle stress responses in maintaining Immuno-metabolic homeostasis in cardiovascular diseases (CVDs)

Charles Yeaman, PhD

  • Molecular mechanisms underlying renal cystogenesis, with a focus on dysfunction of the primary cilia 

Sam M. Young, Jr. PhD

  • Development of gene therapies for hearing restoration/preservation
  • Development of gene therapies to treat neurodegeneration
  • Development of Adenoviral vectors for gene therapy applications
  • Development of gene therapies to treat ataxia