Translation and Application of Gene Editing
December 2015The 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.
Repurposing CRISPR-Cas systems as DNA-based smart antimicrobials
The scope and challenges of visualizing CRISPR-Cas9 gene editing in real time
Current gene editing strategies for the mucopolysaccharidoses
Recent advances in the use of ZFN-mediated gene editing for human gene therapy
In vivo genome editing as a therapeutic strategy for treatment of inherited retinal dystrophies: how close are we to realization?
Gene editing of peripheral blood T cells for cancer immunotherapy
Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy
A Digital PCR-Based Method for Efficient & Highly Specific Screening of Genome Edited Cells
Dr Fyodor Urnov: The power and promise of ZFN-mediated genome editing
Spotlight on: Translation and application of gene editing
The promise of therapeutic genome engineering
Exciting developments in CRISPR/Cas9-mediated approaches for Duchenne MD
Beta testing: preclinical genome editing in β-globin disorders
Stem cell-derived organoid cultures and genome editing tools
Revolutionizing genome editing with CRISPR/Cas9: patent battles and human embryos
Fyodor Urnov: The power & promise of ZFN-mediated genome editing
Access the Gene Editing Resource Center
Learn how Droplet Digital PCR works
Free Webinar hosted by Bio-Rad
Simultaneous Quantification of HDR & NHEJ Alleles by Genome Editing Using ddPCR
Transfecting ZFNs, TALENs or CRISPR/Cas Using Nucleofector™ Technology
Genome Editing using Nucleofector™ Technology – Technical Reference Guide
Transfection of Genome Editing Tools Using Nucleofector™ Technology – Selected Publications
