gestions made. The suggestions are identified with the speaker who made them and should not be construed as reflecting consensus from the workshop or endorsement by the Institute of Medicine.


•    Establish pharmacodynamic biomarkers for early drug efficacy readout because of the difficulty of measuring the target RNA-binding proteins in cerebrospinal fluid or plasma. (Rigo)

•    Establish a clear clinical development path to drug approval because there are no currently approved disease-modifying drugs for many neurodegenerative diseases. (Rigo)

•    Determine how and why RAN proteins are expressed. (Ranum)

•    Identify the roles of RNA versus protein effects in disease. (Ranum)

•    Discern whether RAN translation is a previously unrecognized pathogenic mechanism in neurodegenerative disease. (Ranum)

•    Determine whether neural subtype-specific ncRNA networks contribute to selective regional cellular vulnerabilities. (Mehler)

•    Identify the role of extracellular trafficking of ncRNAs and whether this novel process can lead to local and long-distance propagation of pathology. (Mehler)

The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement