Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.
Cell and gene therapies hold great promise for treating monogenic disorders, yet their preclinical evaluation remains limited by the lack of scalable, human-specific models. Here we establish a patient-derived teratoma xenograft platform as a proof-of-concept system to evaluate ex vivo and in vivo therapeutic strategie...
Mihn Jeong Park, H. Kim, Yuna Hwang et al.
· Experimental and Molecular M... · 0 citations