Former Campbell Lab postdoc Dr. Takako Yoshida-Moriguchi, plays a key role in the DM1 trial
Sanofi-Genzyme is advancing an AAV gene therapy for myotonic dystrophy type 1 (DM1) into a Phase I/II clinical trial. The project, originally initiated by former Campbell Lab postdoctoral fellow Dr. Takako Yoshida-Moriguchi, is now moving forward, with the first patient expected to enroll in late 2025. For additional details, please see the official press release.
Recent Publications:
Willer, T., Inamori, K., Venzke, D., Harvey, C.D., Morgensen, G., Hara, Y., Beltrán Valero de Bernabé, D., Yu, L., Wright, K.M., Campbell, K.P. The glucuronyltransferase B4GAT1 is required for initiation of LARGE-mediated α-dystroglycan functional glycosylation. eLife 3;3:e03941, 2014. [PDF]
Goddeeris, M.M., Wu, B., Venzke, D., Yoshida-Moriguchi, T., Saito, F., Matsumura, K., Moore, S.A., Campbell, K.P. Large glycans on dystroglycan function as a tunable matrix scaffold to prevent dystrophy. Nature 503: 136-40, 2013. [PDF]
Yoshida-Moriguchi, T., Willer, T., Anderson, ME, Venzke, D., Whyte, T., Muntoni, F., Lee, H., Nelson, SF, Yu, L., Campbell, KP. SGK196 is a Glycosylation-Specific O-Mannose Kinase Required for Dystroglycan Function. Science341: 896-9, 2013. [PDF]
Inamori K., Hara Y., Willer T., Anderson M.E., Zhu Z., Yoshida-Moriguchi T., Campbell K.P. Xylosyl- and glucuronyltransferase functions of LARGE in α-dystroglycan modification are conserved in LARGE2. Glycobiology 23: 295-302, 2013. [PDF]
Rojek, J.M., Moraz, M.-L., Pythoud, C., Rothenberger, S., Gisou Van der Goot, F., Campbell, K.P. and Kunz, S. Binding of Lassa virus perturbs extracellular matrix-induced transduction via dystroglycan. Cell Microbiol. 14:1122-1134, 2012. [PDF]
Willer, T., Lee, H., Lommel, M., Yoshida-Moriguchi, T., Beltran Valero de Bernabe, D., Venzke, D., Cirak, S., Schachter, H., Vajsar, J., Voit, T., Muntoni, F., Loder, A.S., Dobyns, W.B., Winder, T.L., Strahl, S., Mathews, K.D., Nelson, S.F., Moore, S.A. and Campbell, K.P. ISPD loss-of-function mutations disrupt dystroglycan O-mannosylation and cause Walker-Warburg syndrome. Nat. Genet. 44: 575-80, 2012. [PDF]
Honors and Awards:
2009
"Campbell Receives March of Dimes Prize in Developmental Biology" go to article
2007
Campbell To Give 2007 Presidential Lecture Feb. 25 go to article
Other News:
On May 6th, 2006 the Campbell Lab participated in a MDA Research Day hosted by the Iowa Center for Muscular Dystrophy Research.