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Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene.
Martínez-Pizarro, Ainhoa; Leal, Fátima; Holm, Lise Lolle; Doktor, Thomas K; Petersen, Ulrika S S; Bueno, María; Thöny, Beat; Pérez, Belén; Andresen, Brage S; Desviat, Lourdes R.
Afiliação
  • Martínez-Pizarro A; Centro de Biología Molecular Severo Ochoa UAM-CSIC, Centro de Diagnóstico de Enfermedades Moleculares (CEDEM), CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.
  • Leal F; Centro de Biología Molecular Severo Ochoa UAM-CSIC, Centro de Diagnóstico de Enfermedades Moleculares (CEDEM), CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.
  • Holm LL; Department of Biochemistry and Molecular Biology, University of Southern Denmark, Odense, Denmark.
  • Doktor TK; Department of Biochemistry and Molecular Biology, University of Southern Denmark, Odense, Denmark.
  • Petersen USS; Department of Biochemistry and Molecular Biology, University of Southern Denmark, Odense, Denmark.
  • Bueno M; Congenital Metabolic Diseases Unit, Hospital Virgen del Rocio, Sevilla, Spain.
  • Thöny B; Division of Metabolism, University Children's Hospital Zürich, Zürich, Switzerland.
  • Pérez B; Centro de Biología Molecular Severo Ochoa UAM-CSIC, Centro de Diagnóstico de Enfermedades Moleculares (CEDEM), CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.
  • Andresen BS; Department of Biochemistry and Molecular Biology, University of Southern Denmark, Odense, Denmark.
  • Desviat LR; Centro de Biología Molecular Severo Ochoa UAM-CSIC, Centro de Diagnóstico de Enfermedades Moleculares (CEDEM), CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.
Nucleic Acid Ther ; 32(5): 378-390, 2022 10.
Article em En | MEDLINE | ID: mdl-35833796
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Sítios de Splice de RNA Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Sítios de Splice de RNA Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article