A novel Pah-exon1 deleted murine model of phenylalanine hydroxylase (PAH) deficiency

Phenylalanine hydroxylase (PAH) deficiency, colloquially known as phenylketonuria (PKU), is among the most common inborn errors of metabolism and in the past decade has become a target for the development of novel therapeutics such as gene therapy. PAH deficient mouse models have been key to new tre...

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Detaylı Bibliyografya
Yayımlandı:Mol Genet Metab
Asıl Yazarlar: Richards, Daelyn Y., Winn, Shelley R., Dudley, Sandra, Fedorov, Lev, Rimann, Nicole, Thöny, Beat, Harding, Cary O.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2020
Konular:
Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC8173763/
https://ncbi.nlm.nih.gov/pubmed/33051130
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2020.09.005
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