Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.

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Published in Mol Ther on September 28, 2010

Authors

Carmen Unzu1, Ana Sampedro, Itsaso Mauleón, Manuel Alegre, Stuart G Beattie, Rafael Enríquez de Salamanca, Jolanda Snapper, Jaap Twisk, Harald Petry, Gloria González-Aseguinolaza, Julio Artieda, María Sol Rodríguez-Pena, Jesús Prieto, Antonio Fontanellas

Author Affiliations

1: Gene Therapy and Hepatology Area, Centre for Applied Medical Research, University of Navarra, Pamplona, Spain.

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