Key References
- 3E10Fv Review Paper:
Antibody mediated transduction of therapeutic proteins into living cells.
Hansen et al. The Scientific World Journal (2005) vol: 5, pp.782-788.
- ENT2 Transport Identification:
Intranuclear Protein Transduction through a Nucleoside Salvage Pathway.
Hansen et al. JBC (2007) vol. 282, no. 29, pp. 20790–20793.
- Myotubular Myopathy Rationale (Viral-mediated replacement previously demonstrated):
AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis.
Buj-bello et al. Hum Mol Genet (2008) vol.17, no. 14, pp. 2132-43.
- Myotonic Dystrophy Rationale (Viral-mediated replacement previously demonstrated):
Reversal of RNA missplicing and myotonia after muscleblind over-expression in a mouse poly(CUG) model for myotonic dystrophy.
Kanadia et al. PNAS (2006) vol. 103, no. 31.