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

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.

Name thesis:degree_name

Master of Sciences

Level thesis:degree_level

masters

Discipline thesis:degree_discipline

Biology

Grantor dc:publisher

Case Western Reserve University School of Graduate Studies

Year dc:date

2013

Author dc:creator

• Davis, Rohit Michael

Contributors dc:contributor

• Cullis, Christopher
• Ritzmann, Roy

• Biology
• Genetics
• Health Care
• Marketing
• Pharmaceuticals
• Muscular Dystrophy
• Follistatin
• Market feasibility
• Commercialization
• Orthopedic application

Statement dc:rights

• unrestricted
• This thesis or dissertation is protected by copyright: some rights reserved. It is licensed for use under a Creative Commons license. Specific terms and permissions are available from this document's record in the OhioLINK ETD Center.

Language dc:language

English

Repository record dc:identifier

http://rave.ohiolink.edu/etdc/view?acc_num=case1346854771

OAI identifier oai:identifier

oai:etd.ohiolink.edu:case1346854771