The Nicolau lab aims to develop gene therapies for inherited neuromuscular disorders, with an emphasis on gene editing. Our goal is to leverage recently developed gene editing techniques to correct mutations causing neuromuscular disease. The design of several of our gene editing systems was informed by patients seen in our clinic, as well as by genetic variants seen in asymptomatic individuals. In addition, we seek to investigate and overcome obstacles to efficient in vivo muscle gene editing, including muscle regeneration, immune reactions, and accuracy of the gene editing. Our lab works with cell culture models, including transdifferentiated patient cells, as well as with several mouse models of muscular dystrophy. In these experiments, we make heavy use of molecular cloning, AAV production, automated quantitative immunofluorescence analysis, capillary western immunoassays, droplet digital PCR, and long-read sequencing.