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Tuesday, October 15, 2013

Molecular Therapy, Volume 21, Issue 10, Pages 1807-1969

Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease
Maria do Carmo Costa, Katiuska Luna-Cancalon, Svetlana Fischer, Naila S Ashraf, Michelle Ouyang, Rahil M Dharia, Lucas Martin-Fishman, Yemen Yang, Vikram G Shakkottai, Beverly L Davidson, Edgardo Rodríguez-Lebrón and Henry L Paulson
Mol Ther 21: 1898-1908; advance online publication, August 6, 2013; doi:10.1038/mt.2013.144

Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease MTOpen
Ana Sergijenko, Alexander Langford-Smith, Ai Y Liao, Claire E Pickford, John McDermott, Gabriel Nowinski, Kia J Langford-Smith, Catherine LR Merry, Simon A Jones, J Edmond Wraith, Robert F Wynn, Fiona L Wilkinson and Brian W Bigger
Mol Ther 21: 1938-1949; advance online publication, July 16, 2013; doi:10.1038/mt.2013.141

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