Courtney Young

Courtney Young

Home Area: Cell and Developmental Biology
Mentor: Melissa Spencer
Research project:

Courtney’s research focus is to develop a CRISPR/Cas9-mediated gene editing therapy for Duchenne muscular dystrophy.


Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy. Young CS, Mokhonova E, Quinonez M, Pyle AD, Spencer MJ. J Neuromuscul Dis. 2017;4(2):139-145. doi: 10.3233/JND-170218.

Exon Skipping Therapy. Young CS, Pyle AD. Cell. 2016 Nov 17;167(5):1144. doi: 10.1016/j.cell.2016.10.050.

CRISPR/Cas9-mediated correction of the sickle mutation in human CD34+ cells. Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, Hollis RP, Kohn DB. Mol Ther. 2016 Jul 13. doi: 10.1038/mt.2016.148. [Epub ahead of print]

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. Cell Stem Cell. 2016 Apr 7;18(4):533-40. doi: 10.1016/j.stem.2016.01.021. Epub 2016 Feb 11.