Eleven percent intact PGM3 in a severely immunodeficient patient with a novel splice-site mutation, a case report by Karin E. Lundin, Qing Wang, Abdulrahman Hamasy, Per Marits, Mehmet Uzunel, Valtteri Wirta, Ann-Charlotte Wikström, Anders Fasth, Olov Ekwall, C.I. Edvard Smith

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Repeatable, Inducible Micro-RNA-Based Technology Tightly Controls Liver Transgene Expression by Iulian I Oprea, Joana R Viola, Pedro M D Moreno, Oscar E Simonson, Sergey Rodin, Nathalie Teller, Karl Tryggvason, Karin E Lundin, Leonard Girnita, Carl Inge Edvard Smith

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Lipophilic Peptide Dendrimers for Delivery of Splice-Switching Oligonucleotides by Haneen Daralnakhla, Osama Saher, Susanna Zamolo, Safa Bazaz, Jeremy P. Bost, Marc Heitz, Karin E. Lundin, Samir EL Andaloussi, Tamis Darbre, Jean-Louis Reymond, Rula Zain, C. I. Edvard Smith

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Micro-minicircle Gene Therapy: Implications of Size on Fermentation, Complexation, Shearing Resistance, and Expression by Sofia Stenler, Oscar PB Wiklander, Maria Badal-Tejedor, Janne Turunen, Joel Z Nordin, David Hallengärd, Britta Wahren, Samir EL Andaloussi, Mark W Rutland, CI Edvard Smith, Karin E Lundin, Pontus Blomberg

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