Pathway for Approval of a Gene Therapy Orphan Product:Treading New Ground FREE
Barry
J Byrne
Mol Ther
21: 1465-1466; doi:10.1038/mt.2013.157
Minicircle DNA is Superior to Plasmid DNA in ElicitingAntigen-specific CD8+ T-cell Responses
Wynette
M Dietz, Nicole E B Skinner, Sara E Hamilton, Michelle
D Jund, Suzanne M Heitfeld, Adam J Litterman, Patrick Hwu,
Zhi-Ying Chen, Andres M Salazar, John R Ohlfest, Bruce
R Blazar, Christopher A Pennell and Mark J Osborn
Mol Ther
21: 1526-1535; advance online publication, May 21, 2013; doi:10.1038/mt.2013.85
AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7snRNA-mediated Exon-skipping Therapy
Maëva Le
Hir, Aurélie Goyenvalle, Cécile Peccate, Guillaume Précigout,
Kay E Davies, Thomas Voit, Luis Garcia and Stéphanie Lorain
Mol Ther
21: 1551-1558; advance online publication, June 11, 2013; doi:10.1038/mt.2013.121
Codelivery of VEGF siRNA and Gemcitabine Monophosphate in aSingle Nanoparticle Formulation for Effective Treatment of NSCLC
Yuan Zhang,
Nicole MJ Schwerbrock, Arlin B Rogers, William Y Kim and
Leaf Huang
Mol Ther
21: 1559-1569; advance online publication, June 18, 2013;
doi:10.1038/mt.2013.120
Biodegradable Lipids Enabling Rapidly Eliminated LipidNanoparticles for Systemic Delivery of RNAi Therapeutics
Martin
A Maier, Muthusamy Jayaraman, Shigeo Matsuda, Ju Liu,
Scott Barros, William Querbes, Ying K Tam, Steven M Ansell,
Varun Kumar, June Qin, Xuemei Zhang, Qianfan Wang,
Sue Panesar, Renta Hutabarat, Mary Carioto,
Julia Hettinger, Pachamuthu Kandasamy, David Butler,
Kallanthottathil G Rajeev, Bo Pang, Klaus Charisse, Kevin Fitzgerald,
Barbara L Mui, Xinyao Du, Pieter Cullis, Thomas D Madden,
Michael J Hope, Muthiah Manoharan and Akin Akinc
Mol Ther
21: 1570-1578; advance online publication, June 25, 2013;
doi:10.1038/mt.2013.124
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