Horizon Discovery, a global leader in gene editing and gene
modulation technologies, in partnership with The Michael J. Fox Foundation for
Parkinson's Research (MJFF), today introduced two new gene knock-out and
knock-in rat models for investigating the role of alpha-synuclein in brain
function and Parkinson’s disease (PD) pathogenesis.
Innovative and
relevant models for understanding alpha-synuclein biology and Parkinson’s
disease pathogenesis in vivo
Alpha-synuclein (SNCA) is a major
constituent of Lewy bodies, protein clumps that are the pathological hallmark
of PD, and so is an important target for research into the onset and
development of the disease. To support this work, Horizon has used its SAGEspeed® Custom Model Generation Platform to
develop and launch two innovative models with genetic modifications of SNCA,
creating both a knock-out of endogenous alpha-synuclein and a humanized A53T
knock-in, which has been linked to early-onset PD. The A53T knock-in promises
to be field-enabling. As the only model available that expresses humanized A53T
alpha-synuclein driven by the endogenous promoter without endogenous rat
alpha-synuclein expression, it is expected to provide unambiguous insights into
the biological effects of the A53T mutation in human alpha-synuclein.
This project builds on Horizon’s
long-standing partnership with MJFF that aims to provide the most innovative
and relevant pre-clinical models to the neuroscience research community. In addition,
the availability of these two new rat models meets a key objective of the MJFF
Parkinson’s Research Tools Consortium (www.michaeljfox.org/toolsconsortium),
a partnership between MJFF and industry groups to develop new tools to address
unmet challenges in PD research. Tools such as the new models from Horizon and
MJFF meet critical needs for both academic and industry experts by deepening
understanding of alpha-synuclein biology and PD etiology, and advancing
treatments for Parkinson’s disease. MJFF is leveraging Horizon’s Sponsored
Breeding Program to help ensure that these models are widely available to the
broader research community, including both for-profit and not-for-profit
organizations, at an affordable price.
Previously, Horizon and MJFF have
collaborated on the development of rat models containing mutations in
leucine-rich repeat kinase 2 (LRRK2), Parkin (Park2), PTEN induced putative
kinase 1 (PINK1) and DJ-1 (Park7), all of which have been implicated as
causative for PD. These lines, which are currently being used in studies across
the globe, have been extensively characterized. Phenotype data can be found on
MJFF’s website, as well as in the many publications available on the Horizon
website.
Dr. Darrin M Disley, Chief Executive
Officer, Horizon Discovery, commented: “Animal models, particularly rat given
its more physiologically relevant neurobiology, play a key role in
understanding the molecular mechanisms that govern brain function and the
development of neurological disease. We are deeply gratified to be able to
continue to partner with the Michael J. Fox Foundation, deploying our leading
gene editing platform to develop specialized in vivo disease models as an
important part of their ground-breaking work.”
Nicole Polinski, PhD, associate
director of research programs at The Michael J. Fox Foundation said:
“Researchers rely on advanced research tools and pre-clinical models to
understand underlying causes and mechanisms of Parkinson's disease and to
develop effective treatments. When such tools are unavailable, investigators
must often make their own, investing time and resources that would be better spent
studying PD.The growing collection of in vivo models from Horizon and The
Michael J. Fox Foundation highlights the ongoing commitment of both
organizations to push the boundaries of science, advance Parkinson’s disease
understanding and speed therapeutic breakthroughs.”
For more information about the
alpha-synuclein ratmodels: www.horizondiscovery.com/alpha-synuclein-knockout-rat and www.horizondiscovery.com/alpha-synuclein-a53t-knock-in-rat
https://www.cambridgenetwork.co.uk/news/horizon-partners-michael-j-fox-foundation-parkinsons/
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