Curative gene therapies for rare diseases

por Maldonado, Rocio, Jalil, Sami, Wartiovaara, Kirmo
Publicado 2020

Simultaneous high-efficiency base editing and reprogramming of patient fibroblasts

por Jalil, Sami, Keskinen, Timo, Maldonado, Rocío, Sokka, Joonas, Trokovic, Ras, Otonkoski, Timo, Wartiovaara, Kirmo
Publicado 2021

CRISPR correction of the Finnish ornithine delta-aminotransferase mutation restores metabolic homeostasis in iPSC from patients with gyrate atrophy

por Maldonado, Rocio, Jalil, Sami, Keskinen, Timo, Nieminen, Anni I., Hyvönen, Mervi E., Lapatto, Risto, Wartiovaara, Kirmo
Publicado 2022

Remodeling of the Actin/Spectrin Membrane-associated Periodic Skeleton, Growth Cone Collapse and F-Actin Decrease during Axonal Degeneration

por Unsain, Nicolas, Bordenave, Martin D., Martinez, Gaby F., Jalil, Sami, von Bilderling, Catalina, Barabas, Federico M., Masullo, Luciano A., Johnstone, Aaron D., Barker, Philip A., Bisbal, Mariano, Stefani, Fernando D., Cáceres, Alfredo O.
Publicado 2018

Author Correction: Remodeling of the Actin/Spectrin Membrane-associated Periodic Skeleton, Growth Cone Collapse and F-Actin Decrease during Axonal Degeneration

por Unsain, Nicolas, Bordenave, Martin D., Martinez, Gaby F., Jalil, Sami, von Bilderling, Catalina, Barabas, Federico M., Masullo, Luciano A., Johnstone, Aaron D., Barker, Philip A., Bisbal, Mariano, Stefani, Fernando D., Cáceres, Alfredo O.
Publicado 2018