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dcleveland@ucsd.edu; clagier-tourenne@mgh.harvard.edu
Q.Z., J.J., S.D.C., C.L.-T. and D.W.C. designed research; Q.Z., J.J., T.F.G., A.R.L.S., H.X., L.P., J.R., S.D.C., C.L.-T. and D.W.C analyzed the data; Q.Z., J.J., T.F.G., M.M.D., L.J., A.T., S.D.G., S.G.D., M.J.R., P.K., and Y.Z. performed research; Q.Z., J.J., S.D.C., C.L.-T. and D.W.C. wrote the manuscript.
We thank Brian Myers, Marcus Maldonado, Jeesun Kim, Jaisen Lim, Jean Yasis, Drs. Charles J. Heyser, Dara Ditsworth, Kent Osborn, Jeannie Chew for their advice and technical assistance. We thank Drs. Martin Fugere and Brian Kaspar at AveXis for providing help in sorting mouse ESC-derived motor neurons. We thank Ionis Pharmaceuticals for providing ASOs. We thank all members of the D.W.C., C.L.-T., J.R. and S.D.C. groups for critical suggestions on this project. We apologize to those whose prior work we have not been able to cite in order to comply with editorial limit on the number of citations.
D.W.C. is a consultant for Ionis Pharmaceuticals. The other authors report no conflict of interest.
This work was supported by grants from NINDS/NIH R01-NS27036 to D.W.C. and S.D.C. and R01-NS087227 to C.L.-T.; from the NIA/NIH-supported UCSD Alzheimer’s Disease Research Center to C.L.-T. and D.W.C.; from Target ALS to C.L.-T. and J.R.; from NINDS/NIH R35-NS097273, P01-NS084974 and R01-NS088689 to L.P.; from P01-NS099114 to T.G. and L.P.; from Target ALS to T.G., L.P. and Y.Z.. C.L.-T is the recipient of the Healey Family ALS Endowed Chair for Research. Q.Z. was recipient of a Milton Safenowitz Postdoctoral fellowship and a STARTER grant from the ALS Association. J.J. was recipient of a Career Development grant from the Muscular Dystrophy Association (MDA #479769).