Intrathecal gene therapy rescues a model of demyelinating peripheral neuropathy

Inherited demyelinating peripheral neuropathies are progressive incurable diseases without effective treatment. To develop a gene therapy approach targeting myelinating Schwann cells that can be translatable, we delivered a lentiviral vector using a single lumbar intrathecal injection and a myelin-s...

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Publicado en:Proc Natl Acad Sci U S A
Autores principales: Kagiava, Alexia, Sargiannidou, Irene, Theophilidis, George, Karaiskos, Christos, Richter, Jan, Bashiardes, Stavros, Schiza, Natasa, Nearchou, Marianna, Christodoulou, Christina, Scherer, Steven S., Kleopa, Kleopas A.
Formato: Artigo
Lenguaje:Inglês
Publicado: National Academy of Sciences 2016
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PNAS Plus
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4855595/
https://ncbi.nlm.nih.gov/pubmed/27035961
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1522202113
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