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

Hyperornithinemia with gyrate atrophy of the choroid and retina (HOGA) is a severe recessive inherited disease, causing muscular degeneration and retinochoroidal atrophy that progresses to blindness. HOGA arises from mutations in the ornithine aminotransferase (OAT) gene, and nearly one-third of the...

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Detalles Bibliográficos
Autores principales: Maldonado, Rocio, Jalil, Sami, Keskinen, Timo, Nieminen, Anni I., Hyvönen, Mervi E., Lapatto, Risto, Wartiovaara, Kirmo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9248217/
https://www.ncbi.nlm.nih.gov/pubmed/35782600
http://dx.doi.org/10.1016/j.ymgmr.2022.100863

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