Translation and Application of Gene Editing

December 2015

The rapidly advancing developments in gene editing have numerous implications and applications for the cell and gene therapy field – from disease modelling through to the potential editing of patient-derived cells to modify/correct disorders.

The Gene Editing spotlight features a lead review from Prof. Andrew Bassett, Oxford University, UK discussing the main gene-editing technologies in detail: Zinc Fingers, TALENs and CRISPR-Cas9, comparing mechanisms of action, limitations and opportunities for future development and applications.

Building upon this, we present a series of Expert Insight articles discussing potential clinical applications in more detail including:

  • Cystic fibrosis
  • Duchenne Muscular Dystrophy
  • Genetic blood disorders and hemoglobinopathies

Alongside the discussion of the incredible advances in gene editing applications, an ongoing patent dispute plays out in the media over the CRISPR-Cas9 technology. Dr Beatriz San Martin, Partner, FieldFisher, UK helps demystify and explain the IP situation and talks about the possible implications of the outcome of this dispute.

We also hear from gene editing industry leaders, Sangamo BioSciences, as Dr Fyodor Urnov discusses their exciting progress towards the clinic with zinc finger nucleases in our exclusive podcast interview.

Spotlight Gene Editing Resource Center

Translation and Application of Gene Editing


Repurposing CRISPR-Cas systems as DNA-based smart antimicrobials

Article Gene Editing Resource Center

The scope and challenges of visualizing CRISPR-Cas9 gene editing in real time


Current gene editing strategies for the mucopolysaccharidoses

Article Gene Editing Resource Center

Recent advances in the use of ZFN-mediated gene editing for human gene therapy

Article Gene Editing Resource Center

In vivo genome editing as a therapeutic strategy for treatment of inherited retinal dystrophies: how close are we to realization?

Article Gene Editing Resource Center

Gene editing of peripheral blood T cells for cancer immunotherapy

Article Gene Editing Resource Center

Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy

Research Article

A Digital PCR-Based Method for Efficient & Highly Specific Screening of Genome Edited Cells

News Audio Podcast - listen now

Dr Fyodor Urnov: The power and promise of ZFN-mediated genome editing

Article Spotlight Foreword

Spotlight on: Translation and application of gene editing

Article Promise of gene editing

The promise of therapeutic genome engineering

Article Gene editing DMD

Exciting developments in CRISPR/Cas9-mediated approaches for Duchenne MD

Article Gene editing beta globin disorders

Beta testing: preclinical genome editing in β-globin disorders

Article Expert Insight Andersson and Koo

Stem cell-derived organoid cultures and genome editing tools

Article Gene Editing Expert Insight

Revolutionizing genome editing with CRISPR/Cas9: patent battles and human embryos

Article Dr Fyodor Urnov Interview

Fyodor Urnov: The power & promise of ZFN-mediated genome editing

Resources Gene Editing Resource Center

Access the Gene Editing Resource Center

Video Learn how Droplet Digital PCR works

Learn how Droplet Digital PCR works

Video Free Webinar hosted by Bio-Rad

Free Webinar hosted by Bio-Rad

ASHG 2015 Poster Simultaneous Quantification of HDR and NHEJ Alleles Induced by Genome Editing Using ddPCR

Simultaneous Quantification of HDR & NHEJ Alleles by Genome Editing Using ddPCR

Webinar View archived webinar

Transfecting ZFNs, TALENs or CRISPR/Cas Using Nucleofector™ Technology

Technical Reference Download Technical Reference Guide

Genome Editing using Nucleofector™ Technology – Technical Reference Guide


Transfection of Genome Editing Tools Using Nucleofector™ Technology – Selected Publications


Access the Lonza Gene Editing Resource Center