Evaluation of orally administered PEGylated phenylalanine ammonia lyase in mice for the treatment of Phenylketonuria

Phenylketonuria (PKU), a Mendelian autosomal recessive phenotype (OMIM 261600), is an inborn error of metabolism causing impaired postnatal cognitive development in the absence of treatment. We used the Pah(enu2/enu2) PKU mouse model to study oral enzyme substitution therapy with various chemically...

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Autores principales: Sarkissian, Christineh N., Kang, Tse Siang, Gámez, Alejandra, Scriver, Charles R., Stevens, Raymond C.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2011
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3205297/
https://ncbi.nlm.nih.gov/pubmed/21803624
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.06.016
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