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Gene Therapy

TABLE OF CONTENTS

Volume 24, Issue 5 (May 2017)

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Delivering efficient liver-directed AAV-mediated gene therapy

J Baruteau, S N Waddington, I E Alexander and P Gissen

Gene Ther 2017 24: 263-264; advance online publication, January 12, 2017; 10.1038/gt.2016.90

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Original Articles

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Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia

D L Ouellet, K Cherif, J Rousseau and J P Tremblay

Gene Ther 2017 24: 265-274; advance online publication, December 26, 2016; 10.1038/gt.2016.89

Abstract | Full Text

An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21/ mouse model

M Perdomini, C Dos Santos, C Goumeaux, V Blouin and P Bougnères

Gene Ther 2017 24: 275-281; advance online publication, February 6, 2017; 10.1038/gt.2017.10

Abstract | Full Text

Live-cell imaging to compare the transfection and gene silencing efficiency of calcium phosphate nanoparticles and a liposomal transfection agent OPEN

S Chernousova and M Epple

Gene Ther 2017 24: 282-289; advance online publication, March 9, 2017; 10.1038/gt.2017.13

Abstract | Full Text

Optimization of adeno-associated virus vector-mediated gene transfer to the respiratory tract

F Kurosaki, R Uchibori, N Mato, Y Sehara, Y Saga, M Urabe, H Mizukami, Y Sugiyama and A Kume

Gene Ther 2017 24: 290-297; advance online publication, March 27, 2017; 10.1038/gt.2017.19

Abstract | Full Text

Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

D Hoffmann, J W Schott, F K Geis, L Lange, F-J Müller, D Lenz, D Zychlinski, D Steinemann, M Morgan, T Moritz and A Schambach

Gene Ther 2017 24: 298-307; advance online publication, April 20, 2017; 10.1038/gt.2017.20

Abstract | Full Text

AAV9-mediated engineering of autotransplanted kidney of non-human primates

S Tomasoni, P Trionfini, N Azzollini, L Zentilin, M Giacca, S Aiello, L Longaretti, E Cozzi, N Baldan, G Remuzzi and A Benigni

Gene Ther 2017 24: 308-313; advance online publication, March 27, 2017; 10.1038/gt.2017.21

Abstract | Full Text

MnSOD mediated by HSV vectors in the periaqueductal gray suppresses morphine withdrawal in rats

T Iida, H Yi, S Liu, D Ikegami, W Zheng, Q Liu, K Takahashi, Y Kashiwagi, W F Goins, J C Glorioso and S Hao

Gene Ther 2017 24: 314-324; advance online publication, April 3, 2017; 10.1038/gt.2017.22

Abstract | Full Text

Short Communication

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Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders

K Bey, C Ciron, L Dubreil, J Deniaud, M Ledevin, J Cristini, V Blouin, P Aubourg and M-A Colle

Gene Ther 2017 24: 325-332; advance online publication, April 20, 2017; 10.1038/gt.2017.18

Abstract | Full Text

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