Selected publications

Genetic and functional correction of argininosuccinate lyase deficiency using CRISPR adenine base editors

Sami Jalil, Timo Keskinen, Juhana Juutila, Rocio Sartori Maldonado, Liliya Euro, Anu Suomalainen, Risto Lapatto, Emilia Kuuluvainen, Ville Hietakangas, Timo Otonkoski, Mervi E Hyvönen, Kirmo Wartiovaara.

The American Journal of Human Genetics, 111: 714-728, 2024

Thymidylate synthase disruption to limit cell proliferation in cell therapies

Rocio Sartori-Maldonado, Hossam Montaser, Inkeri Soppa, Solja Eurola, Juhana Juutila, Melanie Balaz, Henri Puttonen, Timo Otonkoski, Jonna Saarimäki-Vire, Kirmo Wartiovaara.

Molecular Therapy 32: 2535-2548, 2024

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

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

Molecular Genetics and Metabolism Reports