Accepted for Publication: May 1, 2012.
Published Online: November 12, 2012. doi:10.1001/jamaneurol.2013.593
Author Contributions: Mss Winer and Chun and Mr Srinivasan contributed equally to the present work. Study concept and design: Chun, Holtzman, Bennett, Cudkowicz, and Miller. Acquisition of data: Winer, Srinivasan, Chun, Lacomis, Jaffa, Fagan, Wancewicz, Bennett, and Bowser. Analysis and interpretation of data: Winer, Srinivasan, Fagan, Wancewicz, Bennett, Bowser, and Miller. Drafting of the manuscript: Winer, Chun, Jaffa, Bowser, and Miller. Critical revision of the manuscript for important intellectual content: Winer, Srinivasan, Lacomis, Fagan, Holtzman, Wancewicz, Bennett, Bowser, Cudkowicz, and Miller. Statistical analysis: Winer, Srinivasan, and Miller. Obtained funding: Bowser and Miller. Administrative, technical, and material support: Winer, Chun, Lacomis, Fagan, Holtzman, and Miller. Study supervision: Winer, Bennett, Bowser, Cudkowicz, and Miller.
Conflict of Interest Disclosures: Dr Fagan is supported by grants P01 AG03991, P01 AG026276, and U01 AG032438 from the National Institute of Aging of the National Institutes of Health and by the Hope Center for Neurological Disorders. She is a member of the Alzheimer's Disease CSF Biomarker Development Advisory Board for Roche and the US Alzheimer's Disease Advisory Board for Lilly USA. Isis Pharmaceuticals provided the antisense oligonucleotides used in this study.
Funding/Support: This work was supported by an ALS Association pilot grant (to Dr Miller) and Clinical and Translation Science Award grants UL1 RR024992 (to Dr Miller) and K08NS074194 (to Dr Miller). The CSF samples were from Northeast ALS Consortium and the Washington University Knight Alzheimer's Disease Research Center (grants P50-AG05681 [to Dr Morris], P01-AG03991 [to Dr Morris], P01-AG26276 [to Dr Morris], and P30-NS057105 [to Dr Holtzman]), the Charles and Joanne Knight Alzheimer's Disease Research Initiative (to Dr Morris), and the University of Pittsburgh (grants RO1 NS061867-01 [to Dr Bowser] and RC1 NS068179-01 [to Dr Bowser])
Additional Contributions: We thank John C. Morris, MD, for a critical reading of the manuscript and for providing clinical samples.