Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.

Zhou, Haiyan; Meng, Jinhong; Malerba, Alberto; Catapano, Francesco; Sintusek, Palittiya; Jarmin, Susan; Feng, Lucy; Lu-Nguyen, Ngoc; Sun, Lianwen; Mariot, Virginie; Dumonceaux, Julie; E Morgan, Jennifer; Gissen, Paul; Dickson, George; Muntoni, Francesco

doi:10.1002/jcsm.12542

Public

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.

Shared by NobleBlocks on Jun 1, 2020 • 12:00 AM UTC

Authors:

Haiyan Zhou

Jinhong Meng

Alberto Malerba

Abstract

BACKGROUND: Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1) gene that lead to SMN deficiency. Different SMN-restoring therapies substantially prolong survival and function in transgenic mice of SMA. However, these therapies do not entirely prevent mus...

Subject

Adeno-associated virus

Antisense oligonucleotide

Combinatorial therapy

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