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pro vyhledávání: '"Cathryn S Mah"'
Autor:
Darin J Falk, Meghan S Soustek, Adrian Gary Todd, Cathryn S Mah, Denise A Cloutier, Jeffry S Kelley, Nathalie Clement, David D Fuller, Barry J Byrne
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)
Pompe disease is an autosomal recessive genetic disorder characterized by a deficiency of the enzyme responsible for degradation of lysosomal glycogen (acid α-glucosidase (GAA)). Cardiac dysfunction and respiratory muscle weakness are primary feat
Externí odkaz:
https://doaj.org/article/442ce2508cf241e9bda6b41c905115b6
Autor:
Melissa A. Lewis, Thomas J. Conlon, Denise E. Cloutier, Alice F. Tarantal, Irene Zolotukhin, Barry J. Byrne, Christina A. Pacak, Cathryn S. Mah, Bijoy Thattaliyath
Publikováno v:
Circulation research. 99(4)
Heart disease is often the end result of inherited genetic defects, which may potentially be treatable using a gene-transfer approach. Recombinant adeno-associated virus (rAAV)-mediated gene delivery has emerged as a realistic method for the treatmen