Item Details

title

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

author

Guan, Xuan

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

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

type

Dissertation