Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

Patients with mucopolysaccharidosis type I (MPS I), a genetic deficiency of the lysosomal enzyme α-l-iduronidase (IDUA), exhibit accumulation of glycosaminoglycans in tissues, with resulting diverse clinical manifestations including neurological, ocular, skeletal, and cardiac disease. MPS I is curre...

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Detalhes bibliográficos
Main Authors: Hinderer, Christian, Bell, Peter, Gurda, Brittney L., Wang, Qiang, Louboutin, Jean-Pierre, Zhu, Yanqing, Bagel, Jessica, O’Donnell, Patricia, Sikora, Tracey, Ruane, Therese, Wang, Ping, Haskins, Mark E., Wilson, James M.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2014
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4205647/
https://ncbi.nlm.nih.gov/pubmed/25267637
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1413645111
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