Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Phillip A. Doerfler"'
Autor:
David D. Fuller, Amy Poirier, Barry J. Byrne, Phillip A. Doerfler, Lauren Vaught, Michael D. Sunshine, Brendan M. Doyle, Marda Jorgensen, Darin J. Falk, Sara M.F. Turner
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 194-203 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Pompe disease is caused by mutations in the gene encoding the lysosomal glycogen-metabolizing enzyme, acid-alpha glucosidase (GAA). Tongue myofibers and hypoglossal motoneurons appear to be particularly susceptible in Pompe disease. Here we used intr
Autor:
Darin J. Falk, Roland W. Herzog, Adrian G. Todd, Barry J. Byrne, Phillip A. Doerfler, Sushrusha Nayak, Nathalie Clement
Publikováno v:
Human Gene Therapy. 27:43-59
Pompe disease is a progressive neuromuscular disorder caused by lysosomal accumulation of glycogen from a deficiency in acid alpha-glucosidase (GAA). Replacement of the missing enzyme is available by repeated protein infusions; however, efficacy is l
Publikováno v:
Human gene therapy methods. 25(5)
Limiting factors in large preclinical and clinical studies utilizing adeno-associated virus (AAV) for gene therapy are focused on the restrictive packaging capacity, the overall yields, and the versatility of the production methods for single AAV vec
Autor:
Barry J. Byrne, Sushrusha Nayak, Phillip A. Doerfler, Laurence Morel, Manuela Corti, Roland W. Herzog
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA) deficiency). Unfortunately, the introduction of GAA and viral vectors encoding the enzyme can lead
Autor:
Phillip A. Doerfler, Nathalie Clement, Adrian G. Todd, Darin J. Falk, Roland W. Herzog, Barry J. Byrne
Publikováno v:
Molecular Therapy. 23:S146
Enzyme replacement therapy (ERT) for Pompe disease (acid α-glucosidase deficiency; GAA) provokes the development of high titer antibodies against the enzyme and contributes to life threatening infusion associated reactions. AAV-mediated gene therapy
Autor:
E Lavassani, Manuela Corti, Nathalie Clement, Lee Ann Lawson, Kirsten E. Erger, Darin J. Falk, Thomas J. Conlon, Barry J. Byrne, Sushrusha Nayak, Barbara K. Smith, Phillip A. Doerfler, Melissa E. Elder, Roland W. Herzog, MM Green
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Gene therapy strategies for congenital myopathies may require repeat administration of adeno-associated viral (AAV) vectors due to aspects of the clinical application, such as: (i) administration of doses below therapeutic efficacy in patients enroll