Optimizing gene therapy for infant mice with citrullinemia type 1
Citrullinemia type 1 (CTLN1) is a rare genetic disorder that is inherited in an autosomal recessive manner. It is caused by mutations in the gene that encodes argininosuccinate synthetase 1 (ASS1), which catalyzes the formation of argininosuccinate from citrulline and the aspartic acid in the ure...
| Main Authors: | , , |
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| Format: | info:eu-repo/semantics/doctoralThesis |
| Language: | eng |
| Published: |
Universidad de Navarra
2023
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| Subjects: | |
| Online Access: | https://hdl.handle.net/10171/67875 |