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

• Main Authors: Tai-Heng Chen (Author), Shih-Hsin Chang (Author), Yu-Fu Wu (Author), Ya-Ping Yen (Author), Fang-Yu Hsu (Author), Yen-Chung Chen (Author), Yang Ming (Author), Ho-Chiang Hsu (Author), Yi-Ching Su (Author), Sheng-Tang Wong (Author), Jui-Hung Hung (Author), Shih-Hwa Chiou (Author), Yuh-Jyh Jong (Author), Jun-An Chen (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 oligonucleotide treatment is effective for ameliorati...