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Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts.
Buratti, Emanuele; Peruzzo, Paolo; Braga, Luca; Zanin, Irene; Stuani, Cristiana; Goina, Elisa; Romano, Maurizio; Giacca, Mauro; Dardis, Andrea.
Affiliation
  • Buratti E; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Padriciano, Trieste, Italy.
  • Peruzzo P; Centre for Rare Diseases, Academic Hospital Santa Maria della Misericordia, Udine, Italy.
  • Braga L; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Padriciano, Trieste, Italy.
  • Zanin I; Department of Life Sciences, Via Valerio 28, University of Trieste, 34127 Trieste, Italy.
  • Stuani C; Centre for Rare Diseases, Academic Hospital Santa Maria della Misericordia, Udine, Italy.
  • Goina E; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Padriciano, Trieste, Italy.
  • Romano M; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Padriciano, Trieste, Italy.
  • Giacca M; Department of Life Sciences, Via Valerio 28, University of Trieste, 34127 Trieste, Italy.
  • Dardis A; International Centre for Genetic Engineering and Biotechnology (ICGEB), Area Science Park, Padriciano, Trieste, Italy.
Mol Ther Methods Clin Dev ; 20: 227-236, 2021 Mar 12.
Article in En | MEDLINE | ID: mdl-33426149
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Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2021 Type: Article Affiliation country: Italy
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2021 Type: Article Affiliation country: Italy