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Gene Therapy in Combination with Nitrogen Scavenger Pretreatment Corrects Biochemical and Behavioral Abnormalities of Infant Citrullinemia Type 1 Mice

Citrullinemia type I (CTLN1) is a rare autosomal recessive disorder caused by mutations in the gene encoding argininosuccinate synthetase 1 (ASS1) that catalyzes the third step of the urea cycle. CTLN1 patients suffer from impaired elimination of nitrogen, which leads to neurotoxic levels of circula...

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Detalles Bibliográficos
Autores principales:	Bazo, Andrea, Lantero, Aquilino, Mauleón, Itsaso, Neri, Leire, Poms, Martin, Häberle, Johannes, Ricobaraza, Ana, Bénichou, Bernard, Combal, Jean-Philippe, Gonzalez-Aseguinolaza, Gloria, Aldabe, Rafael
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9736988/ https://www.ncbi.nlm.nih.gov/pubmed/36499263 http://dx.doi.org/10.3390/ijms232314940

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