(gentle acoustic music)
- My name is Justin Reynolds.
I live in Jackson, Michigan,
and I teach high school English.
Guess my background with HD
really began regarding a test that my dad
had gone through,
talking about that he had tested positive.
And so I made the decision after I turned 18.
I waited a couple of years.
I think I got tested when I was 20
and I tested positive.
I do have the HD gene.
I'm so interested in learning about the clinical approaches
that we have to finding a cure for HD,
because that's what we're all here for.
Together, hoping for the same outcome.
- So we have a lot of things that usually we do
in the lab tour about the disease,
how we study it,
and we have some activities usually with the visitors.
So it's more interactive.
And so we'll probably, can do that,
and Bhairavi will lead the tour.
And we can ask any question if you have any question,
but that's probably a nice day.
- Research has always been this thing, given provided,
fed, whatever you wanna say to families.
to help with the heartache.
Yes, we have to deal with all this
but there's a light at the end of the tunnel
and it's this piece.
We're gonna get to it by this research aspect specifically.
- We need to have you have a lab coat
- [Justin] I need a coat.
- And because it's inside,
everybody will have to have goggles and gloves.
That's the requirement to be in the lab.
We do something very simple to visualize,
'cause a lot people don't realize what DNA actually is.
So we have a very simple experiment
that we do with visitors,
with banana, because bananas, they got DNA.
And we can just visualize the banana of the DNA.
Also that help us to talk more about the disease,
knowing the gene and the mutation on the gene.
It's actually on this tiny piece of DNA.
I think the science is getting there more and more, now.
We have tools that can really help,
way better than before
'cause before we're just working more
on the cause of the disease.
Now we can really act on really the gene,
which the gene, you remove the gene,
you cannot remove the disease.
- So the first step is to take a small piece of,
maybe you can take half of it,
and kind of mush it in this bag, yeah, that's good.
Make sure you don't have any lumps,
so it should be a consistency of a pudding.
So basically what's happening is you have salt in here.
So when you have, when you add the hot water with salt,
basically the osmosis that happens.
So once that is done, we add the salt water
to the bag of banana mush.
- [Justin] Salt water.
- [Bhairavi] Yep, okay.
Then we squeeze it for about 30 to 45 seconds.
- [Justin] 30, 45.
- So you extracted DNA of banana, congratulations.
- Yes.
- That's the DNA.
What is important is that, this is a food,
but we all our body are made of DNA.
And so that's really the thing with Huntington disease,
there is one specific gene
and one specific in the DNA that has a mutation.
And so that's really what we are studying.
The new technology that we have,
it's really all about working with the gene itself,
because we know in the genes where it is,
we know now for 30 years, where is the gene.
We know the exact structure of the gene.
- We also know the location.
- The location and everything.
With the new technology with CRISPR cas9,
we can cut the gene and repair it.
So that's what the research is really focusing on now,
on the, really the DNA editing tools.
- Probably the most exciting thing I think is gene editing.
I think that gives a lot of hope
for a very effective treatment down the line.
And that's the big thing right now is to be able
to target it to the right,
you don't want to knock
down all of the good Huntington protein.
- Right, 'cause it's there,
and some of it's working.
It's just the bad part.
- You want to get rid of
as much of the bad Huntington protein as you can.
So that's really the key.
- I think the biggest shock for me throughout the tour
was just seeing that this process is a time consuming piece.
You gotta have patience for the process.
- So after the cell culture and working with the cells,
this is really one thing that we do a lot
and Bhairavi study all the form of this brain.
We work with the animal, we inject the drugs, we inject DNA.
We inject any treatment that we believe could help.
We work with the animal.
Just to look at their behavior, if that improve or not.
And at the end we collect their brain.
- [Bhairavi] That is a full mouse brain.
- [Justin] That's the full, okay.
- [Bhairavi] Yeah, that's a full mouse.
That's all the size from our brain.
It's very small.
- We collect those and just the brain.
And after the nice thing, we just use this equipment
to slice the brain very thin slice.
- We collect this specific region of the brain
and then we put them into a liquid
that has, you can see the slices floating.
We're using these nano particles
which have the ability to get the treatment to the brain
when we inject them via blood.
We will be able to stain these slices
and then image them under the microscope
to see the drugs that we have given, or the DNA,
the genetic editing tools,
like where they are and what it is doing to the neurons.
So the red dots that you see here
are the neurons in the mouse brain.
So this one is stained in red color
and will be, say, for example,
will have the DNA that we are putting into the brain
same green color or any other color.
Then we'll be able to see both
at the same time to see, okay,
the treatment that we gave is actually
in the region that we want.
- [Justin] And these are the ones impacted with-
- Yes, these are called the medium spiny neurons
that are impacted in Huntington's disease.
- So that's give you kind of an idea really
of what we do every day with a lot of students here at CMU.
After, if you have any question
we'll be more than happy, with Bhairavi,
myself and Gary, to answer that.
- One thing I would say to all HD patients
is that, don't give up hope.
We are working very hard and our students
are working very hard
and we're just one lab of many around the world, right?
We can get every day, a foot closer and a foot closer.
And I really think that we're on the precipice,
that we're gonna have what is close enough
to be a cure for this disease.
- Today's tour has definitely left me optimistic
that we have the right steps in place.
This is gonna be something that
will lead to the answer and
I'll help pass that message along too.
(gentle acoustic music)