Project Title: In Vivo Genome Editing with the CRISPR/Cas9 System Using Novel Muscle-Tropic AAV Vectors
Aravind Asokan, PhD, Associate Professor, UNC School of Medicine
Charles Gersbach, PhD, Assistant Professor, Duke Pratt School of Engineering
This project aims to develop a gene therapy for Duchenne Muscular Dystrophy (DMD). The investigators are developing a strategy for in vivo delivery of CRISPR using novel adeno-associated virus (AAV) vectors developed for muscle-specific gene delivery.
Project Title: CFTR Therapeutics for Heart Disease: Repurposing Established Drugs and Identifying New Ones
Martina Gentzsch, PhD, Assistant Professor, UNC School of Medicine
Dawn Bowles, PhD, Assistant Professor, Duke School of Medicine
This project aims to develop a cardiac model for testing the feasibility of repurposing cystic fibrosis drugs for treating cardiovascular conditions affected by disruptions in CFTR regulation.
Project Title: Sildenafil Exposure and Safety in Premature Infants
Daniel Gonzalez, PharmD, PhD, Assistant Professor, UNC Eshelman School of Pharmacy
Christoph Hornik, MD, MPH, Assistant Professor, Duke School of Medicine
Sildenafil is often used in premature infants despite limited safety data. Attempts at large prospective clinical trials have been unsuccessful in premature infants due small size, pharmacokinetic variability, and lack of clear safety signals. This project proposes to combine data from a previously conducted pharmacokinetic trial of sildenafil in premature infants with extensive clinical data obtained from a large multicenter electronic medical record based clinical database. Using a previously developed pharmacokinetic model, we will predict sildenafil exposure in infants and evaluate its association with safety events.
Project Title: High-throughput in vitro physiology for the human intestinal epithelium
Scott Magness, PhD, Associate Professor, UNC School of Medicine
John Rawls, PhD, Associate Professor, Duke School of Medicine
This project aims to develop a high-throughput gut-on-a-chip that will enable mechanistic studies of epithelial physiology using human ‘mini-guts’ (a.k.a enteroids). This data will inform the understanding the intestinal mechanisms behind diseases such as inflammatory bowel disease, intestinal failure, obesity and malnutrition, and enables greater understanding of potential therapeutic targets.