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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.
Konkle, Barbara A; Walsh, Christopher E; Escobar, Miguel A; Josephson, Neil C; Young, Guy; von Drygalski, Annette; McPhee, Scott W J; Samulski, R Jude; Bilic, Ivan; de la Rosa, Maurus; Reipert, Birgit M; Rottensteiner, Hanspeter; Scheiflinger, Friedrich; Chapin, John C; Ewenstein, Bruce; Monahan, Paul E.
Afiliação
  • Konkle BA; BloodWorks Northwest, Seattle, WA.
  • Walsh CE; Division of Hematology, University of Washington School of Medicine, Seattle, WA.
  • Escobar MA; Icahn School of Medicine at Mount Sinai, New York, NY.
  • Josephson NC; McGovern Medical School and Gulf States Hemophilia and Thrombophilia Center, University of Texas Health Science Center Houston Medical School, Houston, TX.
  • Young G; Division of Hematology, University of Washington School of Medicine, Seattle, WA.
  • von Drygalski A; Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA.
  • McPhee SWJ; Hemophilia and Thrombosis Treatment Center, University of California, San Diego, CA.
  • Samulski RJ; Asklepios BioPharmaceutical and Chatham Therapeutics, Chapel Hill, NC.
  • Bilic I; Gene Therapy Center, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC.
  • de la Rosa M; Baxalta Innovations GmbH, a member of the Takeda group of companies, Vienna, Austria; and.
  • Reipert BM; Baxalta Innovations GmbH, a member of the Takeda group of companies, Vienna, Austria; and.
  • Rottensteiner H; Baxalta Innovations GmbH, a member of the Takeda group of companies, Vienna, Austria; and.
  • Scheiflinger F; Baxalta Innovations GmbH, a member of the Takeda group of companies, Vienna, Austria; and.
  • Chapin JC; Baxalta Innovations GmbH, a member of the Takeda group of companies, Vienna, Austria; and.
  • Ewenstein B; Baxalta US Inc., a member of the Takeda group of companies, Cambridge, MA.
  • Monahan PE; Baxalta US Inc., a member of the Takeda group of companies, Cambridge, MA.
Blood ; 137(6): 763-774, 2021 02 11.
Article em En | MEDLINE | ID: mdl-33067633
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas Recombinantes de Fusão / Fator IX / Terapia Genética / Regulação da Expressão Gênica / Hemofilia B / Ilhas de CpG Tipo de estudo: Clinical_trials / Etiology_studies / Observational_studies / Risk_factors_studies Limite: Adolescent / Adult / Aged / Humans / Male / Middle aged Idioma: En Revista: Blood Ano de publicação: 2021 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: Proteínas Recombinantes de Fusão / Fator IX / Terapia Genética / Regulação da Expressão Gênica / Hemofilia B / Ilhas de CpG Tipo de estudo: Clinical_trials / Etiology_studies / Observational_studies / Risk_factors_studies Limite: Adolescent / Adult / Aged / Humans / Male / Middle aged Idioma: En Revista: Blood Ano de publicação: 2021 Tipo de documento: Article