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Article

DG9-Conjugated Morpholino Rescues Phenotype in Spinal Muscular Atrophy Mice.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2026

Haque Umme Sabrina, Kohut Melissa, Yokota Toshifumi

Abstract excerpt

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease characterized by decreased expression of the survival of motor neuron 1 (SMN1) gene. Antisense oligonucleotide (ASO)-based therapies have been developed recently as a means of increasing the production of functional SMN protein by targeting the SMN2 gene. Nusinersen, an FDA-approved ASO, has shown success in SMA treatment. However, it faces...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 41028674
DOI: 10.1007/978-1-0716-4738-7_2

Genes

SMN2
SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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