Corporate Banner
Satellite Banner
Scientific Community
Become a Member | Sign in
Home>News>This Article

Horizon and UCL Institute of Neurology Collaborate in rAAV-mediated Genome Editing

Published: Friday, November 09, 2012
Last Updated: Thursday, November 08, 2012
Bookmark and Share
Program will focus on developing human isogenic cell lines modeling Huntington’s disease.

Horizon Discovery (Horizon) has announced that it has entered into a research collaboration for gene editing at the Institute of Neurology, University College London (UCL).

The program of research will focus on the creation of human isogenic disease model cell lines with insertion of Huntington’s disease-causing triplet repeats.

The UCL collaboration contributes to an academic not-for-profit research group to which Horizon commits resources to provide training and access to its proprietary rAAV-mediated human gene-editing platform, GENESIS™.

The program at UCL represents a new disease area for Horizon, and the insertion of triplet repeats into a wild type genome is a novel genetic alteration application for the GENESIS technology.

Prof. Sarah Tabrizi, Professor of Clinical Neurology at the UCL Institute of Neurology, global Principal Investigator of the TRACK-HD study and leader of all UCL Huntington’s disease projects, said: “Although mouse models have given important insights, it is important when studying human disease to use a broad approach, including cultured cells. The ability to accurately introduce the triplet repeat mutation into human somatic cell lines offers exciting possibilities in the study of Huntington’s disease and potential therapies.”

“The use of patient-relevant disease models created by Horizon’s rAAV-mediated genome editing technology is well established in oncology,” commented Dr Rob Howes, Principal Scientist, Horizon Discovery.

Dr Howes continued, “We believe that by developing the application of this technology to other disease areas such as Huntington’s disease, we can provide a vital tool for understanding, preventing and treating those diseases.”

The new human isogenic cell lines generated by UCL will be exclusively licensed to Horizon.  Horizon will also have an exclusive option to license new intellectual property developed.

This forms part of Horizon’s strategy to generate at least 2500 new X-MAN™ (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease.

These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalize many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

The UCL collaboration will complement the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the National Cancer Institute, Cambridge University, Yale University, and Dana-Farber Cancer Institute.

Horizon recently launched an online support site,, for scientists working with rAAV-mediated genome editing.

Further Information
Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 2,600+ scientific posters on ePosters
  • More than 3,800+ scientific videos on LabTube
  • 35 community eNewsletters

Sign In

Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Related Content

Horizon Enters Bioproduction Cell Line Licensing Agreement with LakePharma, Inc.
LakePharma to take a full commercial license for GS null CHO K1 cell line from Horizon for improved efficiency in biopharmaceutical manufacturing.
Wednesday, September 16, 2015
Horizon and Redx Sign Cancer Research Collaboration
Collaboration to progress Redx Pharma plc’s novel pan-RAF inhibitor program towards out-licensing.
Friday, September 04, 2015
Horizon Discovery Appoints Mr Grahame Cook as a Non-Executive Director
Grahame brings over 20 years’ experience to Horizon.
Thursday, May 14, 2015
Transgenomic to Incorporate Horizon's Genomic Reference Standards
Horizon to provide human genomic reference standards to assure quality and performance of Transgenomic’s Multiplexed ICE COLD-PCR kits.
Thursday, April 23, 2015
Horizon Discovery & Thermo Agreement
Horizon Discovery Group plc and Thermo Fisher Scientific sign supply and commercialization agreement, which will see Thermo market and distribute Horizon’s X-MAN cell line collection.
Monday, April 20, 2015
Horizon and Partners Awarded Up to £6.2M Funding
Horizon to receive up to £652,000 to expand bioproduction research.
Thursday, March 26, 2015
Horizon Discovery Group plc and ArcherDX Sign OEM Agreement
Horizon’s Reference Materials chosen by ArcherDX to validate its FusionPlex test for non-small cell lung cancer markers.
Tuesday, March 24, 2015
Horizon Discovery Group plc Named Business of the Year at Business Weekly Awards
Horizon joins select group of companies to receive award for a second time.
Wednesday, March 18, 2015
Horizon to Support Cancer Research UK’s Stratified Medicine Program
Horizon’s Diagnostics division to provide genetically defined, human genomic reference standards.
Friday, March 13, 2015
Horizon Discovery Group to Support Cancer Research UK’s Stratified Medicine Program
Horizon’s Diagnostics division to provide genetically defined, human genomic reference standards.
Wednesday, March 11, 2015
Horizon Discovery CEO Darrin Disley wins Entrepreneur of the Year
Darrin Disley, CEO of Horizon Discovery, wins Entrepreneur of the Year award at the Quoted Company Awards 2015.
Thursday, January 29, 2015
Horizon Discovery Group plc Trading Update
Revenues expected to be ahead of consensus by approximately 7% driven by an increased global demand for expanded product, service and IP offering.
Monday, January 19, 2015
Horizon Discovery Group plc Acquires Haplogen Genomics GmbH
Strategic acquisition of high-throughput functional genomics platform aims to drive product development, revenue growth and enable broader market penetration.
Thursday, January 08, 2015
Horizon Discovery CEO Wins Executive of the Year
Darrin Disley wins Executive of the Year award at the SCRIP Awards.
Thursday, December 04, 2014
Horizon Discovery Group Signs Agreement with Top Ten Pharma Company for Approximately $750,000
Horizon CombinatoRx to perform drug combination screening on a lead oncology asset.
Monday, November 17, 2014
Scientific News
Lung Repair and Regeneration Gene Discovered
New role for hedgehog gene offers better understanding of lung disease.
3 Ways Viruses Have Changed Science for the Better
Viruses are really good at what they do, and we’ve been able to harness their skills to learn about – and potentially improve – human health in several ways.
Mixed Up Cell Transportation Key Piece of ALS and Dementia Puzzle
Researchers from the University of Toronto are one step closer to solving this incredibly complex puzzle, offering hope for treatment.
New Gene Therapy for Vision Loss From a Mitochondrial Disease
NIH-funded study shows success in targeting mitochondrial DNA in mice.
Five New Genetic Variants Linked to Brain Cancer Identified
The biggest ever study of DNA from people with glioma – the most common form of brain cancer – has discovered five new genetic variants associated with the disease.
Predictive Model for Breast Cancer Progression
Biomedical engineers have demonstrated a proof-of-principle technique that could give women and their oncologists more personalized information to help them choose options for treating breast cancer.
Fatty Liver Disease and Scarring Have Strong Genetic Component
Researchers say that hepatic fibrosis, which involves scarring of the liver that can result in dysfunction and, in severe cases, cirrhosis and cancer, may be as much a consequence of genetics as environmental factors.
Specific Variations in RNA Splicing Linked to Breast Cancer
Researchers have identified cellular changes that may play a role in converting normal breast cells into tumors. Targeting these changes could potentially lead to therapies for some forms of breast cancer.
Finding Links and Missing Genes
A catalogue of large-scale genetic changes around the world.
Scientists Test New Gene Therapy for Vision Loss from a Mitochondrial Disease
NIH-funded study shows success in targeting mitochondrial DNA in mice.
Skyscraper Banner

Skyscraper Banner
Go to LabTube
Go to eposters
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
2,600+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
3,800+ scientific videos