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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June 22 2017

Review

The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

V Christie-Brown, J Mitchell and K Talbot

Gene Ther advance online publication, June 22, 2017; doi:10.1038/gt.2017.47; accepted article preview online May 31, 2017

Short Communication

Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing

T Friedmann

Gene Ther advance online publication, June 22, 2017; doi:10.1038/gt.2017.48

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June 15 2017

Original Articles

Synergistic effects of deleting multiple nonessential elements in nonreplicative HSV-1 BAC genomic vectors play a critical role in their viability

M Ventosa, A Ortiz-Temprano, H Khalique and F Lim

Gene Ther advance online publication, June 15, 2017; doi:10.1038/gt.2017.43; accepted article preview online May 29, 2017

SOST silencing promotes proliferation and invasion and reduces apoptosis of retinoblastoma cells by activating Wnt/β-catenin signaling pathway

T Wu, L-N Wang, D-R Tang and F-Y Sun

Gene Ther advance online publication, June 15, 2017; doi:10.1038/gt.2017.31

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June 8 2017

Review

Engineering liposomal nanoparticles for targeted gene therapy

C Zylberberg, K Gaskill, S Pasley and S Matosevic

Gene Ther advance online publication, June 8, 2017; doi:10.1038/gt.2017.41; accepted article preview online May 15, 2017

Original Article

Effect of IL-1β, TNF-α and IGF-1 on trans-endothelial passage of synthetic vectors through an in vitro vascular endothelial barrier of striated muscle

J P Gomez, C Gonçalves, C Pichon and P Midoux

Gene Ther advance online publication, June 8, 2017; doi:10.1038/gt.2017.40; accepted article preview online May 15, 2017

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June 1 2017

Reviews

Herpesvirus microRNAs for use in gene therapy immune-evasion strategies

S T F Bots and R C Hoeben

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.37; accepted article preview online May 9, 2017

Cure SMA and our patient community celebrate the first approved drug for SMA

J Glascock, M Lenz, K Hobby and J Jarecki

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.39; accepted article preview online May 15, 2017

Current application of CRISPR/Cas9 gene-editing technique to eradication of HIV/AIDS

Z Huang, A Tomitaka, A Raymond and M Nair

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.35; accepted article preview online May 4, 2017

Original Articles

CD34+ cells from dental pulp stem cells with a ZFN-mediated and homology-driven repair-mediated locus-specific knock-in of an artificial β-globin gene

S Chattong, O Ruangwattanasuk, W Yindeedej, A Setpakdee and K Manotham

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.42; accepted article preview online May 22, 2017

INSR gene polymorphisms correlate with sensitivity to platinum-based chemotherapy and prognosis in patients with epithelial ovarian cancer

J-L Hu, X-L Hu, Q Han, A-Y Guo, C-J Wang, Y-Y Wen and S-D Cang

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.26; accepted article preview online April 24, 2017

Coinjection of IL2 DNA enhances E7-specific antitumor immunity elicited by intravaginal therapeutic HPV DNA vaccination with electroporation

Y Sun, S Peng, A Yang, E Farmer, T-C Wu and C-F Hung

Gene Ther advance online publication, June 1, 2017; doi:10.1038/gt.2017.38; accepted article preview online May 11, 2017

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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

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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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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