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

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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Bibliographic Details
Main Authors: Rocio Maldonado, Sami Jalil, Timo Keskinen, Anni I. Nieminen, Mervi E. Hyvönen, Risto Lapatto, Kirmo Wartiovaara
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
CRISPR/Cas9
gene editing
gyrate atrophy
HOGA OAT
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426922000234

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http://www.sciencedirect.com/science/article/pii/S2214426922000234

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