ADVANCE ONLINE PUBLICATION

The latest research papers, published online ahead of print. These online versions are definitive and may be cited using the digital object identifier (DOI).
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May 25 2017

Reviews

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry

F Mavilio

Gene Ther advance online publication, May 25, 2017; doi:10.1038/gt.2017.36

How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy

N N Singh, M D Howell, E J Androphy and R N Singh

Gene Ther advance online publication, May 25, 2017; doi:10.1038/gt.2017.34; accepted article preview online May 9, 2017

Original Article

Potential mechanisms of microRNA-141-3p to alleviate chronic inflammatory pain by downregulation of downstream target gene HMGB1: in vitro and in vivo studies

W-S Shen, X-Q Xu, N-N Zhai, Z-S Zhou, J Shao and Y-H Yu

Gene Ther advance online publication, May 25, 2017; doi:10.1038/gt.2017.28; accepted article preview online April 25, 2017

Short Communication

Antigen-presenting cell-targeted lentiviral vectors do not support the development of productive T-cell effector responses: implications for in vivo targeted vaccine delivery

C Goyvaerts, Y De Vlaeminck, D Escors, S Lienenklaus, M Keyaerts, G Raes and K Breckpot

Gene Ther advance online publication, May 25, 2017; doi:10.1038/gt.2017.30

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May 11 2017

Reviews

New treatments for serious conditions: ethical implications

N M P King and C E Bishop

Gene Ther advance online publication, May 11, 2017; doi:10.1038/gt.2017.32; accepted article preview online May 3, 2017

Building the patient community

F Raffai and O Timmis

Gene Ther advance online publication, May 11, 2017; doi:10.1038/gt.2017.33; accepted article preview online May 3, 2017

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May 4 2017

Original Articles

Characterization of retroviral infectivity and superinfection resistance during retrovirus-mediated transduction of mammalian cells Open

J Liao, Q Wei, J Fan, Y Zou, D Song, J Liu, F Liu, C Ma, X Hu, L Li, Y Yu, X Qu, L Chen, X Yu, Z Zhang, C Zhao, Z Zeng, R Zhang, S Yan, T Wu, X Wu, Y Shu, J Lei, Y Li, W Zhang, J Wang, R R Reid, M J Lee, W Huang, J M Wolf, T-C He and J Wang

Gene Ther advance online publication, May 4, 2017; doi:10.1038/gt.2017.24

Development of potent class II transactivator gene delivery systems capable of inducing de novo MHC II expression in human cells, in vitro and ex vivo

M L Palma, P Duangkhae, B Douradinha, I F T Viana, P O Rigato, R Dhalia, R B Mailliard, S M Barratt-Boyes, E J M Nascimento, T M Oshiro, A J da Silva Duarte and E T A Marques

Gene Ther advance online publication, May 4, 2017; doi:10.1038/gt.2017.25; accepted article preview online April 17, 2017

Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes

J J Siu, N J Queen, W Huang, F Q Yin, X Liu, C Wang, D M McTigue and L Cao

Gene Ther advance online publication, May 4, 2017; doi:10.1038/gt.2017.27; accepted article preview online April 25, 2017

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March 9 2017

Review

Extracellular vesicles for nucleic acid delivery: progress and prospects for safe RNA-based gene therapy FREE

L Jiang, P Vader and R M Schiffelers

Gene Ther advance online publication, March 9, 2017; doi:10.1038/gt.2017.8; accepted article preview online January 31, 2017

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February 23 2017

Original Article

Adeno-associated virus-mediated expression of myostatin propeptide improves the growth of skeletal muscle and attenuates hyperglycemia in db/db mice FREE

J G Jiang, G F Shen, J Li, C Qiao, B Xiao, H Yan, D W Wang and X Xiao

Gene Ther advance online publication, February 23, 2017; doi:10.1038/gt.2016.85; accepted article preview online December 16, 2016

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February 16 2017

Review

Genetic pharmacology: progresses in siRNA delivery and therapeutic applications FREE

D Scherman, A Rousseau, P Bigey and V Escriou

Gene Ther advance online publication, February 16, 2017; doi:10.1038/gt.2017.6; accepted article preview online January 25, 2017

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February 2 2017

Review

Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems FREE

S Guan and J Rosenecker

Gene Ther advance online publication, February 2, 2017; doi:10.1038/gt.2017.5

Original Article

Evidence for the in vivo safety of insulated foamy viral vectors FREE

D L Browning, E M Everson, D J Leap, J D Hocum, H Wang, G Stamatoyannopoulos and G D Trobridge

Gene Ther advance online publication, February 2, 2017; doi:10.1038/gt.2016.88

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January 19 2017

Original Article

Late responses to adenoviral-mediated transfer of the aquaporin-1 gene for radiation-induced salivary hypofunction FREE

I Alevizos, C Zheng, A P Cotrim, S Liu, L McCullagh, M E Billings, C M Goldsmith, M Tandon, E J Helmerhorst, M A Catalán, S J Danielides, P Perez, N P Nikolov, J A Chiorini, J E Melvin, F G Oppenheim, G G Illei and B J Baum

Gene Ther advance online publication, January 19, 2017; doi:10.1038/gt.2016.87

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January 5 2017

Review

Graphene materials as 2D non-viral gene transfer vector platforms FREE

M Vincent, I de Lázaro and K Kostarelos

Gene Ther advance online publication, January 5, 2017; doi:10.1038/gt.2016.79; accepted article preview online November 22, 2016

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December 1 2016

Review

Non-viral delivery of genome-editing nucleases for gene therapy FREE

M Wang, Z A Glass and Q Xu

Gene Ther advance online publication, December 1, 2016; doi:10.1038/gt.2016.72