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Lipid Nanoparticle mRNA Therapy Improves Survival and Reduces Serum Branched-Chain Amino Acids in Mouse Models of Maple Syrup Urine Disease.
Greig, Jenny A; Jennis, Matthew; Dandekar, Aditya; Chorazeczewski, Joanna K; Param, Nesteene; So, Meardey; Nayal, Mohamad; Bell, Peter; Coughlan, Kimberly; Choi, Minjung; Giangrande, Paloma H; Martini, Paolo G V; Wilson, James M.
Afiliación
  • Greig JA; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Jennis M; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Dandekar A; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Chorazeczewski JK; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Param N; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • So M; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Nayal M; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Bell P; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Coughlan K; Moderna, Inc., Cambridge, Massachusetts, USA.
  • Choi M; Moderna, Inc., Cambridge, Massachusetts, USA.
  • Giangrande PH; Moderna, Inc., Cambridge, Massachusetts, USA.
  • Martini PGV; Moderna, Inc., Cambridge, Massachusetts, USA.
  • Wilson JM; Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Hum Gene Ther ; 35(17-18): 726-733, 2024 Sep.
Article en En | MEDLINE | ID: mdl-39001827
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: ARN Mensajero / Modelos Animales de Enfermedad / Nanopartículas / Aminoácidos de Cadena Ramificada / Enfermedad de la Orina de Jarabe de Arce Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: ARN Mensajero / Modelos Animales de Enfermedad / Nanopartículas / Aminoácidos de Cadena Ramificada / Enfermedad de la Orina de Jarabe de Arce Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos