ACCEPTED ARTICLE PREVIEW

Latest accepted manuscripts for publication in Gene Therapy.
These manuscripts will undergo copyediting, typesetting and a proof review before being published in their final form.
About accepted article preview

Sort articles by article type
Top

June 23 2017

Review

The clinical landscape for SMA in a new therapeutic era Open

K Talbot and E F Tizzano

Gene Ther doi:10.1038/gt.2017.52; accepted article preview online June 23, 2017

Top

June 16 2017

Original Article

Optimization of design and production strategies for novel adeno-associated viral display peptide libraries

J Körbelin, A Hunger, M Alawi, T Sieber, M Binder and M Trepel

Gene Ther doi:10.1038/gt.2017.51; accepted article preview online June 16, 2017

Top

June 15 2017

Original Article

miRNA mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity

C A Reid, S L Boye, W W Hauswirth and D M Lipinski

Gene Ther doi:10.1038/gt.2017.50; accepted article preview online June 15, 2017

Top

May 31 2017

Review

Therapeutic approaches for spinal muscular atrophy (SMA)

M Scoto, R S Finkel, E Mercuri and F Muntoni

Gene Ther doi:10.1038/gt.2017.45; accepted article preview online May 31, 2017

Top

May 30 2017

Review

Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy

S Jablonka and M Sendtner

Gene Ther doi:10.1038/gt.2017.46; accepted article preview online May 30, 2017

Top

May 29 2017

Original Article

Silencing of HIV-1 by AgoshRNA molecules

E Herrera-Carrillo, A Harwig and B Berkhout

Gene Ther doi:10.1038/gt.2017.44; accepted article preview online May 29, 2017

Top

April 25 2017

Review

The importance of international collaboration for rare diseases research—a European perspective

D Julkowska, C P Austin, C M Cutillo, D Gancberg, C Hager, J Halftermeyer, A H Jonker, L P L Lau, I Norstedt, A Rath, R Schuster, E Simelyte and S van Weely

Gene Ther doi:10.1038/gt.2017.29; accepted article preview online April 25, 2017