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Our Research
BIOMOLECULE
DELIVERY
|
The
future
success
of
cell
and
gene
therapies
relies
on
developing
new
platforms
for
targeted
biomolecule
delivery,
both
inside
and
outside
the
human
body.
The
Miller
lab
is
taking
a
multi-faceted
approach
to
this
problem,
developing
viral
and
non-viral
methods
to
deliver
nucleic acids and proteins.
Project
Highlight:
RNAAV
|
We
are
developing
a
platform
for
tissue-specific
RNA
delivery
derived
from
an
engineered
adeno-associated
virus
system.
The
preliminary
system,
that
we
call
RNAAV
,
is
capable
of
specifically
packaging
RNA
molecules
without
disrupting
the
capsid
function.
O
OH
B
10
4
10
6
10
8
10
10
DNA
(AAV)
RNA
(RNAAV)
Nucleic acid in capsid
T
i
t
e
r (
geno
m
e
s/
ul
)
wild-type
catalytically dead
RNA-packaging
AAV system:
GENOME
EDITING
|
Recent
advances
in
genome
editing
technology
has
catapulted
this
transformative
basic
science
to
the
clinic,
leading
to
established
one-time
curative
therapies
for
devastating
genetic
disease
(Casgevy,
Abengcemeran).
We
continue
to
develop
the
genome
editing
toolkit,
improving
existing
platforms
and
exploring
new
modes
of
genome
editing,
with
a
focus
on
gene-sized
DNA
insertion
technologies.
Project
Highlight:
MRGE
|
We
are
developing
a
platform
for
CRISPR-free
DNA
insertion.
This
platform,
that
we
call
MRGE,
mediates
targeted
gene-size
insertion
into
the
human
genome
with
simple
and
compact
protein-coding
components.
This
approach
could
enable
mutation-agnostic
treatment of genetic disease.
ACT
B
GAPD
H
0
1
2
3
Genomic locu
s
editing e
f
ficiency
(% mKate positive cells
)
donor onl
y
MRGE
2.1
MRGE
2.4
MRGE
2.2
GOI
MRGE
mKate
GOI
TOOLS
AND
TECHNIQUES
|
Our
lab
employs
a
variety
of
methods
to
develop
these
technologies,
including
directed
evolution,
computational
engineering,
de
novo
protein
design,
rational
engineering,
and
combinations
thereof.
Our
engineering
efforts
are
motivated
by
application,
tailoring
tools
with
transformative
end-goals
in
mind
from
the
start.
Current
translational
applications
in
the
laboratory
include
enginered
stem
cell
therapies,
B
cell
reprogramming, and somatic cell genetic disease correction.
See Publications
Funding
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