Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

Grants

Funding Acknowledgements

We thank Patrick King, Clement Ng, Cheyenne Schloffman, Marcus Maldonado, Anh Bui, and Drs. Ricardos Tabet, Kent Osborn, and Nissi Varki for their advice and technical assistance. We thank all members of D.W.C., C.L.-T., and J.R. groups and the teamat Ionis Pharmaceuticals for critical suggestions on this project. This work was supported by the ALS Association (a Neurocollaborative grant to D.W.C., grants to T.F.G. and L.P., and a Milton Safenowitz postdoctoral fellowship to Q.Z.); grants from the NIH (R01-NS088578 to J.R. and D.W.C., R01-NS087227 to C.L.-T., R21-NS089979 to T.F.G., as well as R21-NS084528, R01-NS088689, R01-NS063964, R01-NS077402, and P01-NS084974 to L.P.); the UCSD Alzheimer's Disease Research Center (to C.L.-T.); research project funding from Target ALS to C.L.-T. (13-04827), J.R. (13-44792), and L.P.; the Robert Packard Center for ALS Research at Johns Hopkins (to L.P.); the Mayo Clinic Foundation (to L.P.); a senior clinical investigatorship and a grant from FWO-Vlaanderen to P.V.D.; the Belgian Alzheimer Disease Association (SAO; to P.V.D.); and the European Union's Seventh Framework Programme FP7/2014-2019 under grant agreement no 617198 [DPR-MODELS] (to D.E.). J. J. was supported by NIH postdoctoral fellowships (T32 AG00216 and F32 NS087842). S. Sun is the recipient of career development grants from the NIH (K99 NS091538) and Target ALS. L.D.M. is employed by Janssen Pharmaceutical Companies of Johnson & Johnson. P.J., S.C., J.E., A.W., C.F.B., and F.R. are employees and D.W.C. is a consultant for Ionis Pharmaceuticals. J.R. received salary support from the University of California, San Diego. C.L.-T. and D.W.C. received salary support from the Ludwig Institute for Cancer Research.