Ryan Lee

Higher Degree by Research Candidate

School of Biomedicine

Faculty of Health and Medical Sciences

Duchenne Muscular Dystrophy affects 1 in 5000 live male births, making it the most common inherited muscular disorder in children globally.

My project focuses on developing CRISPR therapeutics targeting exon 45, which would be therapeutically relevant to 9% of DMD patients.

Year	Citation
2025	Chey, Y. C. J., Gierus, L., Lushington, C., Arudkumar, J. C., B. Geiger, A., Staker, L. G., . . . Thomas, P. Q. (2025). Optimal SpCas9- and SaCas9-mediated gene editing by enhancing gRNA transcript levels through scaffold poly-T tract reduction. BMC Genomics, 26(1), 12 pages. DOI Scopus3