Enzyme Replacement Therapy Ameliorates Multiple Symptoms of Murine Homocystinuria

Classical homocystinuria (HCU) is the most common inherited disorder of sulfur amino acid metabolism caused by deficiency in cystathionine beta-synthase (CBS) activity and characterized by severe elevation of homocysteine in blood and tissues. Treatment with dietary methionine restriction is not opt...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Majtan, Tomas, Jones, Wendell, Krijt, Jakub, Park, Insun, Kruger, Warren D., Kožich, Viktor, Bassnett, Steven, Bublil, Erez M., Kraus, Jan P.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910661/
https://ncbi.nlm.nih.gov/pubmed/29398487
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.014
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