Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Enzyme replacement therapy has revolutionized the treatment of the somatic manifestations of lysosomal storage diseases (LSD), although it has been ineffective in treating central nervous system (CNS) manifestations of these disorders. The development of neurotrophic vectors based on novel serotypes...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Mol Ther
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group 2014
Aiheet:
Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429692/
https://ncbi.nlm.nih.gov/pubmed/25027660
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.135
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