Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1.

Kukreja, Anjli; Lasaro, Melissa; Cobaugh, Christian; Forbes, Chris; Tang, Jian-Ping; Gao, Xiang; Martin-Higueras, Cristina; Pey, Angel L; Salido, Eduardo; Sobolov, Susan; Subramanian, Romesh R

Kukreja, Anjli; Lasaro, Melissa; Cobaugh, Christian; Forbes, Chris; Tang, Jian-Ping; Gao, Xiang; Martin-Higueras, Cristina; Pey, Angel L; Salido, Eduardo; Sobolov, Susan; Subramanian, Romesh R.

Afiliación

Kukreja A; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Lasaro M; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Cobaugh C; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Forbes C; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Tang JP; 2 Clinical Pharmacology, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Gao X; 3 Pharmacometrics and Physiologically Based PKPD Modeling and Simulation Clinical Development, Alexion Pharmaceuticals, Inc., Boston, Massachusetts.
Martin-Higueras C; 4 Center for Rare Diseases (CIBERER), Hospital Universitario de Canarias, Universidad de La Laguna, Tenerife, Spain.
Pey AL; 5 Department of Physical Chemistry, Faculty of Sciences, University of Granada, Granada, Spain.
Salido E; 4 Center for Rare Diseases (CIBERER), Hospital Universitario de Canarias, Universidad de La Laguna, Tenerife, Spain.
Sobolov S; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.
Subramanian RR; 1 Research, Alexion Pharmaceuticals, Inc., New Haven, Connecticut.

Nucleic Acid Ther ; 29(2): 104-113, 2019 04.

Article en En | MEDLINE | ID: mdl-30676254

Asunto(s)

Hiperoxaluria Primaria/genética; Hígado/efectos de los fármacos; ARN Mensajero/farmacología; Transaminasas/farmacología; Animales; Modelos Animales de Enfermedad; Vectores Genéticos/genética; Vectores Genéticos/farmacología; Glioxilatos/metabolismo; Humanos; Hiperoxaluria Primaria/terapia; Hígado/metabolismo; Hígado/patología; Ratones; Ratones Noqueados; Oxalatos/metabolismo; ARN Mensajero/genética; Transaminasas/genética

Palabras clave

efficacy; mRNA therapeutic; primary hyperoxaluria type 1

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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Hiperoxaluria Primaria / ARN Mensajero / Transaminasas / Hígado Límite: Animals / Humans Idioma: En Revista: Nucleic Acid Ther Año: 2019 Tipo del documento: Article

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