Treatment of Cystathionine β-Synthase Deficiency in Mice Using a Minicircle-Based Naked DNA Vector

Cystathionine β-synthase (CBS) deficiency is a recessive inborn error of metabolism characterized by extremely elevated total homocysteine (tHcy) in the blood. Patients diagnosed with CBS deficiency have a variety of clinical problems, including dislocated lenses, osteoporosis, cognitive and behavio...

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Publicat a:Hum Gene Ther
Autors principals: Lee, Hyung-Ok, Gallego-Villar, Lorena, Grisch-Chan, Hiu Man, Häberle, Johannes, Thöny, Beat, Kruger, Warren D.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc., publishers 2019
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6761586/
https://ncbi.nlm.nih.gov/pubmed/31084364
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.014
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