Gene Editing
Historical Perspectives Precision Gene Editing with rAAV Vectors Comparison of Gene Editing TechniquesPublications
To date, Horizon has used the rAAV GENESIS™ genome-editing platform to create over 500 X-MAN™ (gene-X Mutant And Normal) isogenic cell lines, the world’s first source of genetically-defined and patient-relevant human cellular models, with more being developed continually. These disease models comprise two cell lines that are identical except for the targeted modification, allowing for the accurate study of normal vs. diseased biology, the identification or validation of novel drug targets, or the identification, development and prescription of targeted or ‘personalized’ medicines.
Horizon provides a range of GENESIS™ and X-MAN™ derived products and services, with clear industrial applications in: bio-pharmaceutical process optimization; clinical diagnostic development; drug discovery & development; and the provision of reference standards for genomics-based clinical research platforms.
Horizon's science base and technology platforms are substantiated by over 400 peer reviewed publications, involvement in 5 multi-national grant-funded translational research consortia, partnering in over 50 academic research collaborations and the establishment of over 30 rAAV human gene-editing Centers of Excellence.
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