Jax Labs eBook - 11

IMPACTFUL SOLUTIONS CREATE INNOVATIVE SCIENCE

years ago, when there were only a
couple of labs who could really do it well
and there was some sort of magic or
secret sauce to the ES cells that only
a couple people knew about. It is quite
user-friendly and, as a result of that,
we have been able as a community to
really invest in making mutations that
perhaps we would not have made before.
Perhaps these are patient-specific
mutations that are not just knockout
but missense mutations, making an
allelic series of mutations, up and down
a particular gene so that we can really
understand what the functionality and
the consequences are.
Those are really powerful tools.
And I would also add that the CRISPR
technology from the vantage point of
making mouse models, is able to be used
in terms of having a therapeutic in which
we are then someday correcting those
mutations in the patient population. The
CRISPR technology is very vast and has a
lot of potential and, as Lizzie alluded to, I
11 | GENengnews.com

think we are only just tapping into it now.
There is a lot more to come.
LEMIEUX: What would you say are some
of the biggest challenges in preclinical
research that are facing the neuro field
right now?
LUTZ: To follow up on the CRISPR and
the different mouse models that are
being made, we are starting to see and
feel that while it is easy to make some of
these mouse models, the phenotyping is
something that requires a lot of care, time
and effort to make sure that we are all
doing it well.
There is some caution to be had when
everybody is making mice faster than
we can possibly phenotype them. It will
introduce some confusion, I am sure, but
it is important to make sure that those
mouse models are referenced and that
people have access to them from various
repositories so that we can follow up on the
published reports and reproduce that data.

There are some challenges in
neurobiology. I refer back to the complex
diseases, such as Alzheimer's and ALS
and Parkinson's disease, where we know
that these are not just genetic diseases.
There is definitely a genetic component
to them, but we do want to make sure
that we do not oversell the science. As
Lizzie said, mice aren't people and people
aren't mice and they are a great tool, but
we have to be cautious to try to not overtranslate what we find in the mice.
We have a tendency to do that
sometimes, in the public eye, where
the thinking is that this model for
the disease is going to answer every
question that we have ever had. And I
don't think that is true. So, some of the
challenges, I think, are about how you
model a complex disease in a mouse.
In neurodegeneration, a lot of these
diseases are also diseases of aging as
well. We are living to our 80s and 90s as
individuals and a population, where these
mice are only living a couple of years.



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Jax Labs eBook

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