{"id":{"repo_id":"ohiolink","oai_identifier":"oai:etd.ohiolink.edu:case1346854771"},"canonical_url":"https://search.dev.ndltd.org/etd/ohiolink/oai:etd.ohiolink.edu:case1346854771","repository":{"repo_id":"ohiolink","name":"OhioLINK","base_url":"https://etd.ohiolink.edu/acprod/odb_etd/ws/oai/oai"},"display":{"title":"THE ASSESSMENT AND DEVELOPMENT OF FOLLISTATIN AS A GENE THERAPY AND ITS POTENTIAL ORTHOPEDIC APPLICATIONS","abstract":"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.","abstract_html":"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.","abstract_has_math":false,"creators":["Davis, Rohit Michael"],"institution":"Case Western Reserve University School of Graduate Studies","degree_name":"Master of Sciences","degree_level":"masters","degree_discipline":"Biology","degree_department":null,"school":null,"contributors":["Cullis, Christopher","Ritzmann, Roy"],"advisors":[],"committee_chairs":[],"committee_members":[],"year":2013,"date_issued":"2013-03-07","date_published":"2013-03-07","updated_at":"2026-07-24T03:35:52Z","subjects":["Biology","Genetics","Health Care","Marketing","Pharmaceuticals","Muscular Dystrophy","Follistatin","Market feasibility","Commercialization","Orthopedic application"],"languages":["English"],"rights":["unrestricted","This thesis or dissertation is protected by copyright: some rights reserved. 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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. 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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. 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