Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS

• Main Authors: Tommaso Iannitti (Author), Joseph M. Scarrott (Author), Shibi Likhite (Author), Ian R.P. Coldicott (Author), Katherine E. Lewis (Author), Paul R. Heath (Author), Adrian Higginbottom (Author), Monika A. Myszczynska (Author), Marta Milo (Author), Guillaume M. Hautbergue (Author), Kathrin Meyer (Author), Brian K. Kaspar (Author), Laura Ferraiuolo (Author), Pamela J. Shaw (Author), Mimoun Azzouz (Author)
• Format: Book
• Published: Elsevier, 2018-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Of familial amyotrophic lateral sclerosis (fALS) cases, 20% are caused by mutations in the gene encoding human cytosolic Cu/Zn superoxide dismutase (hSOD1). Efficient translation of the therapeutic potential of RNAi for the treatment of SOD1-ALS patients requires the development of vectors that are...