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Targeted gene therapy in human-induced pluripotent stem cells from a patient with primary hyperoxaluria type 1 using CRISPR/Cas9 technology.
Estève, Julie; Blouin, Jean-Marc; Lalanne, Magalie; Azzi-Martin, Lamia; Dubus, Pierre; Bidet, Audrey; Harambat, Jérôme; Llanas, Brigitte; Moranvillier, Isabelle; Bedel, Aurélie; Moreau-Gaudry, François; Richard, Emmanuel.
Afiliação
  • Estève J; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Blouin JM; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Lalanne M; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Azzi-Martin L; Univ. Bordeaux, INSERM, Bordeaux Research in Translational Oncology, U1053, CHU Bordeaux, 33076, Bordeaux, France.
  • Dubus P; Univ. Bordeaux, INSERM, Bordeaux Research in Translational Oncology, U1053, CHU Bordeaux, 33076, Bordeaux, France.
  • Bidet A; Laboratoire d'Hématologie, CHU de Bordeaux, Bordeaux, France.
  • Harambat J; Service de Néphrologie Pédiatrique, Centre de Référence Maladies Rénales Rares du Sud-Ouest, CHU Bordeaux, 33000, Bordeaux, France.
  • Llanas B; Service de Néphrologie Pédiatrique, Centre de Référence Maladies Rénales Rares du Sud-Ouest, CHU Bordeaux, 33000, Bordeaux, France.
  • Moranvillier I; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Bedel A; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Moreau-Gaudry F; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France.
  • Richard E; Univ. Bordeaux, INSERM, BMGIC, U1035, CHU Bordeaux, 33076, Bordeaux, France. Electronic address: emmanuel.richard@u-bordeaux.fr.
Biochem Biophys Res Commun ; 517(4): 677-683, 2019 10 01.
Article em En | MEDLINE | ID: mdl-31402115
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Hiperoxalúria Primária / Terapia Genética / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Proteína 9 Associada à CRISPR Limite: Animals / Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Hiperoxalúria Primária / Terapia Genética / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Proteína 9 Associada à CRISPR Limite: Animals / Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article