Gene therapy

MiR34 contributes to spinal muscular atrophy and AAV9-mediated delivery of MiR34a ameliorates the motor deficits in SMA mice

Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by the selective loss of spinal motor neurons (MNs) and concomitant muscle weakness. Mutation of SMN1 is known to cause SMA, and restoring SMN protein levels via antisense …

Mir-17∼92 Confers Motor Neuron Subtype Differential Resistance to ALS-Associated Degeneration

Amyotrophic lateral sclerosis is a neurodegenerative disease that causes progressive paralysis that is lethal and incurable to date. In this article, we discovered a developmentally critical microRNA cluster, _mir-17~92_, plays a role in the vulnerability of spinal motor neurons and supplementing this microRNA cluster can prolong the life expectancy in a mouse disease model.