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Gene Therapy

Analyze biomolecular interactions using AAV biosensors

Prepackaged and Ready-to-use Growing collection of sensors (e.g. Calcium and glutamate) CaMPARI, GCaMP3, GCaMP5, GCaMP6, jRCaMP1, jRGECO1 & iGLuSnFR Choice of promoter and the ability to include Cre inducible (FLEx-ON) expression. Perfect for in vitro or in vivo models.

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Volume 25, Issue 2

In this issue
Brief Communication
Also new
nature.com webcasts

Nature Research
Custom presents a webcast on: MicroRNAs in CSF as prodromal biomarkers for Huntington's disease
Date: Tuesday, May 29, 2018
Join our webcast to learn about the fundamental characteristics of microRNAs and why Huntington's disease may serve as a disease model for the assessment of biomarkers.
This webcast has been produced on behalf of the sponsor who retains sole responsibility for content
Sponsored by: HTG Molecular 


Understanding the molecular biology of intervertebral disc degeneration and potential gene therapy strategies for regeneration: a review

Prasanthi Sampara, Rajkiran Reddy Banala, Satish Kumar Vemuri, Gurava Reddy AV & Subbaiah GPV

Gene Therapy 2018 25 :67 - 82; March 22, 2018; 10.1038/s41434-018-0004-0


Abstract | Full Text


Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector

Melvin Y. Rincon, Filip de Vin, Sandra I. Duqué, Shelly Fripont, Stephanie A. Castaldo et al.

Gene Therapy 2018 25 :83 - 92; March 09, 2018; 10.1038/s41434-018-0005-z


Abstract | Full Text

Brief Communication

Improving therapeutic efficacy of IL-12 intratumoral gene electrotransfer through novel plasmid design and modified parameters

C. Burkart, A. Mukhopadhyay, S. A. Shirley, R. J. Connolly, J. H. Wright et al.

Gene Therapy 2018 25 :93 - 103; March 09, 2018; 10.1038/s41434-018-0006-y


Abstract | Full Text

MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates

Alan H. Nagahara, Bayard R. Wilson, Iryna Ivasyk, Imre Kovacs, Saytam Rawalji et al.

Gene Therapy 2018 25 :104 - 114; March 13, 2018; 10.1038/s41434-018-0010-2


Abstract | Full Text

Evaluation of the Glypican 3 promoter for transcriptional targeting of hepatocellular carcinoma

Bijay Dhungel, Slawomir Andrzejewski, Aparna Jayachandran, Ritu Shrestha, Charmaine A. Ramlogan-Steel et al.

Gene Therapy 2018 25 :115 - 128; March 21, 2018; 10.1038/s41434-018-0002-2


Abstract | Full Text

AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model

Mehmet E. Yalvac, Jakkrit Amornvit, Lei Chen, Kimberly M. Shontz, Sarah Lewis et al.

Gene Therapy 2018 25 :129 - 138; March 09, 2018; 10.1038/s41434-018-0009-8


Abstract | Full Text

Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9

Calvin J. Stephens, Elena Kashentseva, William Everett, Lyudmila Kaliberova & David T. Curiel

Gene Therapy 2018 25 :139 - 156; March 27, 2018; 10.1038/s41434-018-0003-1


Abstract | Full Text

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