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THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS

Davis, Rohit Michael
http://rave.ohiolink.edu/etdc/view?acc_num=case1346854771

Abstract Details

2013, Master of Sciences, Case Western Reserve University, Biology.
Muscular Dystrophies represent a group of inherited disorders that are characterized by muscle weakness and loss of muscle tissue with the symptoms generally worsening over time. Most therapies that have been invented and researched until now have aimed at targeting dystrophin which is the gene that is defective in muscular dystrophy patients. However, there is substantial clinical evidence that the upregulation of follistatin results in better muscle growth and strengthening. Moreover, the use of adeno-associated viruses ensures the efficient, specific delivery of the transgenes to the muscle that requires it. Furthermore, the alternatively spliced variant of Follistatin used in the therapy ensures the prevention of any off-target effects that is a major concern and hurdle in gene therapy development. In this thesis, an attempt has been made to assess the technology for its scientific and commercial feasibility in addition to assessing possible therapeutic applications outside the realm of muscular dystrophies.
Christopher Cullis, Dr. (Advisor)
Jean Welter, Dr. (Committee Member)
Al Hawkins (Committee Member)
Roy Ritzmann, Dr. (Committee Chair)
85 p.
Biology; Genetics; Health Care; Marketing; Pharmaceuticals
Muscular Dystrophy; Follistatin; Market feasibility; Commercialization; Orthopedic application

Recommended Citations

Citations

  • Davis, R. M. (2013). THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS [Master's thesis, Case Western Reserve University]. OhioLINK Electronic Theses and Dissertations Center. http://rave.ohiolink.edu/etdc/view?acc_num=case1346854771

    APA Style (7th edition)

  • Davis, Rohit. THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS. 2013. Case Western Reserve University, Master's thesis. OhioLINK Electronic Theses and Dissertations Center, http://rave.ohiolink.edu/etdc/view?acc_num=case1346854771.

    MLA Style (8th edition)

  • Davis, Rohit. "THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS." Master's thesis, Case Western Reserve University, 2013. http://rave.ohiolink.edu/etdc/view?acc_num=case1346854771

    Chicago Manual of Style (17th edition)

case1346854771
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© 2012, some rights reserved.Creative Commons License THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS by Rohit Michael Davis is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License. Based on a work at etd.ohiolink.edu.
This open access ETD is published by Case Western Reserve University School of Graduate Studies and OhioLINK.