Restoration of the Normal Splicing Pattern of the PLP1 Gene by Means of an Antisense Oligonucleotide Directed against an Exonic Mutation

Open Access

Peer-reviewed

Research Article

Stefano Regis,

Affiliation Centro di Diagnostica Genetica e Biochimica delle Malattie Metaboliche, Istituto G. Gaslini, Genova, Italy
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Fabio Corsolini,

Affiliation Centro di Diagnostica Genetica e Biochimica delle Malattie Metaboliche, Istituto G. Gaslini, Genova, Italy
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Serena Grossi,

Affiliation Centro di Diagnostica Genetica e Biochimica delle Malattie Metaboliche, Istituto G. Gaslini, Genova, Italy
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Barbara Tappino,

Affiliation Centro di Diagnostica Genetica e Biochimica delle Malattie Metaboliche, Istituto G. Gaslini, Genova, Italy
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David N. Cooper,

Affiliation Institute of Medical Genetics, School of Medicine, Cardiff University, Cardiff, United Kingdom
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Mirella Filocamo

* E-mail: mirellafilocamo@ospedale-gaslini.ge.it

Affiliation Centro di Diagnostica Genetica e Biochimica delle Malattie Metaboliche, Istituto G. Gaslini, Genova, Italy
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Transfection
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Morpholino
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Mutation
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Point mutation
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Alternative splicing
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Oligonucleotides
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Reverse transcriptase-polymerase chain reaction
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Antisense oligonucleotides
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