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Advanced filters: Author: Luca Calandriello Clear advanced filters

Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

Spinal cord organoids from SMA patients reveal widespread neurodevelopmental defects. Single-cell and functional analyses show global dysregulation, which can be rescued by SMN-increasing therapy, stressing the importance of timely intervention.

Irene Faravelli
Paola Rinchetti
Stefania Corti
ResearchOpen Access21 Dec 2025
Nature Communications

Volume: 17, P: 1-20
MicroRNA expression analysis identifies a subset of downregulated miRNAs in ALS motor neuron progenitors

Mafalda Rizzuti
Giuseppe Filosa
Stefania Corti
ResearchOpen Access04 Jul 2018
Scientific Reports

Volume: 8, P: 1-12

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Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

MicroRNA expression analysis identifies a subset of downregulated miRNAs in ALS motor neuron progenitors

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