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academies
Cell Science, Stem Cell Research &
Pharmacological Regenerative Medicine
November 29-30, 2017 | Atlanta, USA
Annual Congress on
Adv cel sci tissue cul 2017 | Volume 1 Issue 2
An
ex vivo
gene therapy strategy to treat Duchenne muscular dystrophy using a novel 3D muscle stem
cell culture system and CRISPR/Cas9–mediated genome editing
Wen-Shu Wu
University of Illinois at Chicago, USA
D
uchenne muscular dystrophy (DMD) is a genetic disorder
causedbymutations in
dystrophin
gene and characterized
by progressive muscle degeneration and a shortened life
span and there is no effective treatment available. Muscle
stem cells (MuSCs) exhibit robust myogenic potential
in vivo
,
thus they provide a promising curative treatment for DMD
and other muscle disorders. Because of scarce native adult
MuSCs in muscle tissue and very limited source of biopsies
from patients,
ex-vivo
expansion of freshly isolated MuSCs
is highly desired to achieve a therapeutic cell dose. Here
we develop a soft 3D salmon fibrin gel culture system that
can selectively expand MuSCs from a small number of crude
skeletal muscle cell preparations and faithfully maintain
their regenerative capacity for at least two weeks in culture.
Moreover, we used CRISPR/Cas9-mediated genome editing
to correct the
dystrophin
gene mutation in MuSCs expanded
by this system and restored the skeletal muscle
dystrophin
expression upon transplantation in mdx mice. Our studies
established a reliable and feasible platform for
ex-vivo
expansion and direct gene editing of MuSCs, thus greatly
advancing MuSC-based gene therapies for various muscle
disorders.
Speaker Biography
Wen-Shu Wu has received his Doctoral degree in Cancer Biology from University of
Texas MD Anderson Cancer Center and completed his Post-doctoral training at Dana-
Farber Cancer Institute, Harvard Medical School. He has held faculty positions at
several research institutes. He was an Instructor at Harvard Medical School, a Principal
Investigator at Main Medical Center Research Institute and an Assistant Professor of
the Program in Cell, Molecular and Developmental Biology at Tufts University School
of Medicine. Before joining UIC, he was an Associate Scientist at Children’s Hospital
Oakland Research Institute (CHORI) and a Principal Investigator at the University
of California Berkeley Stem Cell Center. He was recruited to University of Illinois at
Chicago as a tenured Associated Professor in 2013.
e:
ncmishra@mailbox.sc.edu