Rhonda Bassel-Duby

For over two decades my research interests have involved understanding the molecular mechanisms that control striated muscle growth and response to injury and disease.  We have made major contributions in defining the roles of transcription factors, proteins, micropeptides, microRNAs and long non-coding RNAs during striated muscle development and diseases.  Recently, my research has focused on using CRISPR gene editing technology to correct mutations in cardiovascular and skeletal muscle diseases.  We combined induced pluripotent stem cells, animal disease models and CRISPR technologies to develop new therapies for disease.

Publications:    

1. Base editing correction of hypertrophic cardiomyopathy in human cardiomyocytes and humanized mice. Chai AC et al. (2023). Nature Medicine 
2. CRISPR-Editing Therapy for Duchenne Muscular Dystrophy. Chemello F, Olson EN and Bassel-Duby R. (2023). Human Gene Therapy
3. Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease. Lebek S et al. (2023). ​Science​​​​​​
4. Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy. Nishiyama T et al. (2022). Science Translational Medicine
5. Dynamic Transcriptional Responses to Injury of Regenerative and Non-regenerative Cardiomyocytes Revealed by Single-Nucleus RNA Sequencing. Cui M et al. (2020)  Developmental Cell