A combinatorial approach increases SMN level in SMA model mice

Dumas, SA; Villalon, E; Bergman, EM; Wilson, KJ; Marugan, JJ; Lorson, CL; Burnett, BG

Burnett, BG (通讯作者)，Uniformed Serv Univ Hlth Sci, F Edward Hebert Sch Med, Dept Anat Physiol & Genet, Bethesda, MD 20814 USA.;Lorson, CL (通讯作者)，Univ Missouri, Bond Life Sci Ctr, Columbia, MO 65211 USA.;Lorson, CL (通讯作者)，Univ Missouri, Coll Vet Med, Dept Vet Pathobiol, Columbia, MO 65211 USA.

HUMAN MOLECULAR GENETICS, 2022; 31 (17): 2989

Abstract

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by reduced expression of the survival motor neuron (SMN) protein. Current disease-......

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