Bosnakovski, Darko and Nandez, Ramiro and Struck, Matthew and Zaidman, Nathan and Kyba, Michael (2009) Generation of induced pluripotent stem cells from patient-specific FSHD myoblasts. In: 13th Annual Minnesota Muscle Symposium, 9 June 2009, USA.
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Abstract
Generation of induced pluripotent stem cells from patient-specific FSHD
myoblasts
Ramiro Nandez, Darko Bosnakovski, Mathew Struck, Nathan Zaidman and Michael Kyba
Lillehei Heart Institute and Department of Pediatrics, University of Minnesota
Facioscapulohumeral muscular dystrophy (FSHD) is a dominant inherited neuromuscular
disease. The underlying molecular mechanism of the disease is still unknown but 95% of
patients carry a subtelomeric deletion on chromosome 4q35.2 thought to affect chromatin and
gene expression in the region. The recent discovery of methods to reprogram somatic cells to
the embryonic state allows the creation of patient- and disease-specific cells, which can serve
as a model for the disease, and the possibility of autologous cell therapy with genenetically
corrected cells. In this study we have generated 3 induced pluripotent stem (iPS) cell lines
from myoblasts obtained from FSHD patient biopsies as well as a 4th line derived from
unaffected myoblasts. To induce reprogramming we delivered the classical transcription
factor quartet (Oct4, Sox2, Klf4, and Myc) by retroviral transduction and also included SV40
Large-T-antigen to increase the efficiency of reprogramming. These cell lines appear
indistinguishable from human embryonic stem (hES) cells and express hES markers. We are
in the process of characterizing each of the clones for gene expression, teratoma formation
and in vitro differentiation with the intention of understan
| Item Type: | Conference or Workshop Item (Speech) |
|---|---|
| Subjects: | Natural sciences > Biological sciences |
| Divisions: | Faculty of Medical Science |
| Depositing User: | Darko Bosnakovski |
| Date Deposited: | 24 Jan 2014 11:56 |
| Last Modified: | 24 Jan 2014 11:56 |
| URI: | https://eprints.ugd.edu.mk/id/eprint/7052 |
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