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Neuroprotection by Gene Therapy Targeting Mutant SOD1 in Individual Pools of Motor Neurons Does not Translate Into Therapeutic Benefit in fALS Mice

A major challenge in neurological gene therapy is delivery of the transgene to sufficient cell numbers in an atraumatic manner. This is particularly difficult for motor neuron (MN) diseases that have cells located across the entire spinal cord, brain stem, and cortex. We have used the familial mouse...

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Detalhes bibliográficos
Main Authors: Towne, Chris, Setola, Veronica, Schneider, Bernard L, Aebischer, Patrick
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3034853/
https://ncbi.nlm.nih.gov/pubmed/21102563
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.260
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