WEBVTT

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Epithelial cells are the cells
that form the boundary

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between the inside of you
and the outside of you,

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and as such, they have to do two things:

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One is they have to prevent you
from mixing with the outside world,

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but also they have to control
what you take in from the outside world

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and what you excrete
back into the outside world.

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There's a top of the cell
that faces the outside world,

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we call that the apical side,

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and then there are the sides that face the
the intracellular fluid or blood space,

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we call that the basolateral side,

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and then there's glue that holds
those epithelial cells together

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and prevent stuff from leaking between them,

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we call those tight junctions.

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So we're very interested in understanding

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the structures that are
required to mediate function.

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Part of my lab is studying
kidney epithelial cells

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and trying to figure out how they target
proteins to the right places,

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apical versus basolateral,

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and how they organize their unique structures

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in order to carry out their function.

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The rest of the lab is studying
polycystic kidney disease,

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and there are sort of two sets
of projects within that group.

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One is focused on trying to understand
at a molecular and cellular level

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what is it that the proteins encoded
by the polycystic kidney disease genes do?

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What is their physiologic functions?

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And the other part of the lab
is trying to figure out

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what are the therapeutic vulnerabilities
in polycystic kidney disease?

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Are there small molecules
or gene therapy strategies that could be

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used to try and fix the disease,
or at least slow it down?

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We use a lot of different microscopy techniques,

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including immunofluorescence microscopy,

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electron microscopy, to analyze the kidney
and understand its structures.

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We do a lot of protein biochemistry.

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We do a lot of molecular analysis

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and what we're trying to do
is put together a holistic picture

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of what's going on in cells
by interrogating

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all of these different features
that allow us to assess whether

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the pathways and functions
that we're exploring

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and the therapies that we're trying to develop

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mimics the aspects of the human disease.

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Like everybody, we're hoping,
right, that we're going to develop

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a new understanding of how nature
works in the context of how

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epithelial cells organize themselves

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and more immediately,
we're hoping that we can identify

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new mechanisms that are responsible
for polycystic kidney disease

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and, most importantly, new therapies.

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So our lab has developed a couple of therapies,

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one of which is in human clinical trials,

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one of which is hopefully headed
towards human clinical trials.

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The great thing about

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working on a genetic disease
like polycystic kidney disease

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is it can teach us both about

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potential therapies and also
about fundamental physiology.
