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MODELING HEART DISEASE OF PATIENTS WITH MUSCULAR DYSTROPHY USING INDUCED PLURIPOTENT STEM CELLS

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abstract
Dystrophin deficient cardiomyopathy is a hereditary disorder resulting from dystrophin gene mutation. All patients inevitably develop cardiomyopathy and 30%-50% of them succumb to congestive heart failure. The mechanism of dystrophic cardiomyopathy is still elusive, partly due to the scarce human material to study this disease. Therefore, we propose to create dystrophin deficient cardiomyopathy in vitro using DMD patient cardiomyocytes derived from induced pluripotent stem cells (iPSC). By converting adult somatic cells to iPSCs and further differentiate into cardiac lineage, a vast number of cardiomyocytes can be manufactured to explore disease etiology and conduct drug screening.
subject
adeno associated virus
Cellular Reprogramming
Drug Evaluation
Preclinical
Genetic Therapy
Induced Pluripotent Stem Cells
Muscular Dystrophy
Duchenne
contributor
Guan, Xuan (author)
Childers, Martin K (committee chair)
Herrington, David M (committee member)
Christ, George J (committee member)
Furth, Mark E (committee member)
Froehner, Stanley C (committee member)
date
2015-08-25T08:35:32Z (accessioned)
2016-08-24T08:30:10Z (available)
2015 (issued)
degree
Physiology and Pharmacology (discipline)
embargo
2016-08-24 (terms)
identifier
http://hdl.handle.net/10339/57259 (uri)
language
en (iso)
publisher
Wake Forest University
title
MODELING HEART DISEASE OF PATIENTS WITH MUSCULAR DYSTROPHY USING INDUCED PLURIPOTENT STEM CELLS
type
Dissertation

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