Article
Anti-SOD1 Nanobodies That Stabilize Misfolded SOD1 Proteins Also Promote Neurite Outgrowth in Mutant SOD1 Human Neurons.
International journal of molecular sciences - 16 Dec 2022
Kumar Meenakshi Sundaram, Fowler-Magaw Megan E, Kulick Daniel, Boopathy Sivakumar, Gadd Del Hayden, Rotunno Melissa, Douthwright Catherine, Golebiowski Diane, Yusuf Issa, Xu Zuoshang, Brown Robert H, Sena-Esteves Miguel, O'Neil Alison L, Bosco Daryl A
Abstract excerpt
ALS-linked mutations induce aberrant conformations within the SOD1 protein that are thought to underlie the pathogenic mechanism of SOD1-mediated ALS. Although clinical trials are underway for gene silencing of SOD1, these approaches reduce both wild-type and mutated forms of SOD1. Here, we sought to develop anti-SOD1 nanobodies with selectivity for mutant and misfolded forms of human SOD1 over wild-type SOD1....
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