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Arginase I mRNA therapy – a novel approach to rescue arginase 1 enzyme deficiency
Arginase I (ARG1) deficiency is an autosomal recessive urea cycle disorder, caused by deficiency of the enzyme Arginase I, resulting in accumulation of arginine in blood. Current Standard of Care (SOC) for ARG1 deficiency in patients or those having detrimental mutations of ARG1 gene is diet control...
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| Publicado no: | RNA Biol |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Taylor & Francis
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6161738/ https://ncbi.nlm.nih.gov/pubmed/29923457 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/15476286.2018.1475178 |
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