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

• Главные авторы: Tommaso Iannitti (Автор), Joseph M. Scarrott (Автор), Shibi Likhite (Автор), Ian R.P. Coldicott (Автор), Katherine E. Lewis (Автор), Paul R. Heath (Автор), Adrian Higginbottom (Автор), Monika A. Myszczynska (Автор), Marta Milo (Автор), Guillaume M. Hautbergue (Автор), Kathrin Meyer (Автор), Brian K. Kaspar (Автор), Laura Ferraiuolo (Автор), Pamela J. Shaw (Автор), Mimoun Azzouz (Автор)
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• Опубликовано: Elsevier, 2018-09-01T00:00:00Z.
• Предметы: article
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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...