CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis

por Nitzahn, Matthew, Truong, Brian, Khoja, Suhail, Vega-Crespo, Agustin, Le, Colleen, Eliav, Adam, Makris, Georgios, Pyle, April D., Häberle, Johannes, Lipshutz, Gerald S.
Publicado 2021

Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities

por Khoja, Suhail, Lambert, Jenna, Nitzahn, Matthew, Eliav, Adam, Zhang, YuChen, Tamboline, Mikayla, Le, Colleen T., Nasser, Eram, Li, Yunfeng, Patel, Puja, Zhuravka, Irina, Lueptow, Lindsay M., Tkachyova, Ilona, Xu, Shili, Nissim, Itzhak, Schulze, Andreas, Lipshutz, Gerald S.
Publicado 2022