Neurological disease, defined as an abnormal function of the peripheral or central nervous system due to impaired electrical impulses throughout the brain or nervous system, is increasingly common, particularly given an aging population.  In recent years, there has been growing evidence that genetics play a major role in many forms of neurological disease such as Alzheimer’s and Parkinson’s.

Major research efforts have helped us to understand the underlying pathology of many neurological disorders, with the hope of being able to introduce new diagnostic tools and to discover novel targets for new and more effective medications.

We are still in the early days of this research however.  While population approaches have emerged as an indispensable component of genetic research in neurology, identifying many promising biomarkers of disease, much of the work to validate these markers, and to identify new ones, remains. 

Horizon is uniquely suited to helping researchers investigate the underlying basis of disease.  We translate genomic information into practical drug discovery and diagnostic tools that accurately recreate the specific genetics of real patients that are significant in human disease onset and progression.

Horizon’s gene editing platform confers the ability to rapidly introduce any genetic variation into any endogenous gene loci of any human cell line; thus accurately modeling real patient genotypes.

These disease models take the form of cell lines, which are being used widely in basic and drug discovery research and provide them as tools, use them as the basis of a wide range of services to power drug discovery and development programs, and offer them as reference standards to help ensure that patient diagnostic testing provides accurate results. 

A particular strength to support neuroscience research is through Horizon’s in vivo models.  We use our gene editing platform to generate these models, which are powerful tools used to investigate not only the impact of genetic modifications on the cognitive behavior of complex systems, but also to explore whether there are neurological side effects of therapeutics for drugs treating other conditions.



Dong M. et al. (2014). DAG1 mutations associated with asymptomatic hyperCKemia and hypoglycosylation of α-dystroglycan, Neurology. 2015 Jan 20;84(3):273-9

Kuldip D. et al. (2014). Phenotypic characterization of recessive gene knockout rat models of Parkinson's disease, Neurobiology of Disease 70 (2014) 190–203

Shannon M. Hamilton, et al. (2014). Fmr1 and Nlgn3 Knockout Rats: Novel Tools for Investigating Autism Spectrum Disorders, Behavioral Neuroscience 2014, Vol. 128, No. 2, 103–109

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