Leenor Alfahel, Thomas Gschwendtberger, Velina Kozareva, Laura Dumas, Rachel Gibbs, Alexander Kertser, Kuti Baruch, Shir Zaccai, Joy Kahn, Nadine Thau-Habermann, Reto Eggenschwiler, Jared Sterneckert, Andreas Hermann, Niveda Sundararaman, Vineet Vaibhav, Jennifer E Van Eyk, Victor F Rafuse, Ernest Fraenkel, Tobias Cantz, Susanne Petri, Adrian Israelson
Mutations in SOD1 cause amyotrophic lateral sclerosis (ALS), a neurodegenerative disease characterized by motor neuron (MN) loss. We previously discovered that macrophage migration inhibitory factor (MIF), whose levels are extremely low in spinal MNs, inhibits mutant SOD1 misfolding and toxicity. In this study, we show that a single peripheral injection of adeno-associated virus (AAV) delivering MIF into adult SOD1G37R mice significantly improves their motor function, delays disease progression, and extends survival...
April 29, 2024: Cell reports medicine