Production of a heterozygous exon skipping model of common marmosets using gene-editing technology.
Journal
Lab animal
ISSN: 1548-4475
Titre abrégé: Lab Anim (NY)
Pays: United States
ID NLM: 0417737
Informations de publication
Date de publication:
30 Aug 2024
30 Aug 2024
Historique:
received:
10
07
2023
accepted:
29
07
2024
medline:
31
8
2024
pubmed:
31
8
2024
entrez:
30
8
2024
Statut:
aheadofprint
Résumé
Nonhuman primates (NHPs), which are closely related to humans, are useful in biomedical research, and an increasing number of NHP disease models have been reported using gene editing. However, many disease-related genes cause perinatal death when manipulated homozygously by gene editing. In addition, NHP resources, which are limited, should be efficiently used. Here, to address these issues, we developed a method of introducing heterozygous genetic modifications into common marmosets by combining Platinum transcription activator-like effector nuclease (TALEN) and a gene-editing strategy in oocytes. We succeeded in introducing the heterozygous exon 9 deletion mutation in the presenilin 1 gene, which causes familial Alzheimer's disease in humans, using this technology. As a result, we obtained animals with the expected genotypes and confirmed several Alzheimer's disease-related biochemical changes. This study suggests that highly efficient heterozygosity-oriented gene editing is possible using TALEN and oocytes and is an effective method for producing genetically modified animals.
Identifiants
pubmed: 39215182
doi: 10.1038/s41684-024-01424-0
pii: 10.1038/s41684-024-01424-0
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Japan Agency for Medical Research and Development (AMED)
ID : JP20dm0207065
Organisme : Japan Agency for Medical Research and Development (AMED)
ID : 24zf0127007h0003
Organisme : Japan Agency for Medical Research and Development (AMED)
ID : JP20dm0207001
Organisme : MEXT | Japan Society for the Promotion of Science (JSPS)
ID : JP21H04756
Informations de copyright
© 2024. The Author(s).
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