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Article

Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Mar 2022

Bersani Margherita, Rizzuti Mafalda, Pagliari Elisa, Garbellini Manuela, Saccomanno Domenica, Moulton Hong M, Bresolin Nereo, Comi Giacomo P, Corti Stefania, Nizzardo Monica

Abstract excerpt

Spinal muscular atrophy (SMA) is a motor neuron disease and the leading genetic cause of infant mortality. Recently approved SMA therapies have transformed a deadly disease into a survivable one, but these compounds show a wide spectrum of clinical response and effective rescue only in the early stages of the disease. Therefore, safe, symptomatic-suitable, non-invasive treatments with high clinical impact across...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Oligonucleotides, Antisense

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Identifiers

PMID: 34808387
DOI: 10.1016/j.ymthe.2021.11.012
PMCID: PMC8899506

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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