A framework for individualized splice-switching oligonucleotide therapy

por Kim, Jinkuk, Woo, Sijae, de Gusmao, Claudio M., Zhao, Boxun, Chin, Diana H., DiDonato, Renata L., Nguyen, Minh A., Nakayama, Tojo, Hu, Chunguang April, Soucy, Aubrie, Kuniholm, Ashley, Thornton, Jennifer Karlin, Riccardi, Olivia, Friedman, Danielle A., El Achkar, Christelle Moufawad, Dash, Zane, Cornelissen, Laura, Donado, Carolina, Faour, Kamli N. W., Bush, Lynn W., Suslovitch, Victoria, Lentucci, Claudia, Park, Peter J., Lee, Eunjung Alice, Patterson, Al, Philippakis, Anthony A., Margus, Brad, Berde, Charles B., Yu, Timothy W.
Publicado 2023

Metformin for treatment of cytopenias in children and young adults with Fanconi anemia

por Pollard, Jessica A., Furutani, Elissa, Liu, Shanshan, Esrick, Erica, Cohen, Laurie E., Bledsoe, Jacob, Liu, Chih-Wei, Lu, Kun, de Haro, Maria Jose Ramirez, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S., Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria, Boulad, Farid, Carroll, Clint, Hartung, Helge, Hont, Amy, Nakano, Taizo, Olson, Tim, Sze, Sei-Gyung, Thompson, Alexis A., Wlodarski, Marcin W., Gu, Xuesong, Libermann, Towia A., D’Andrea, Alan, Grompe, Markus, Weller, Edie, Shimamura, Akiko
Publicado 2022