29 March 2017
Vol 9, Issue 383
  • Perspective

  • Research Articles

    • Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis

      Poly(GP) proteins are a promising pharmacodynamic marker for developing and testing therapeutics for treating C9ORF72-associated amyotrophic lateral sclerosis.

    • The druggable genome and support for target identification and validation in drug development

      The druggable genome and genome-wide association study data reveal new drug development and repurposing opportunities.

  • Report

  • Editors' Choice

About The Cover

Cover image expansion

ONLINE COVER A Home Run for ALS Research. Amyotrophic lateral sclerosis (ALS), a devastating disease involving progressive motor neuron degeneration, is also called Lou Gehrig's disease after the baseball star (pictured) who died of the illness in 1941. The most common cause of ALS is repeat expansion mutations in the C9ORF72 gene leading to accumulation of G4C2 RNAs and the poly(GP) proteins they encode. Poly(GP) proteins can be detected in the cerebrospinal fluid of ALS patients carrying C9ORF72 mutations and may be a useful pharmacodynamic marker for testing the efficacy of RNA therapies that are entering clinical trials (Gendron et al.). [CREDIT: LOUIS VAN OEYEN/WRHS/CONTRIBUTOR]