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Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model.
Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Aliu, Ermal; Shi, Huifang; Basu, Shakuntala; Kochersperger, Catherine; Van't Land, Clinton; Karunanidhi, Anuradha; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry.
Afiliación
  • Zhao XJ; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Mohsen AW; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Mihalik S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Solo K; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Aliu E; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Shi H; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Basu S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Kochersperger C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Van't Land C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Karunanidhi A; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA.
  • Coughlan KA; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Siddiqui S; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Rice LM; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Hillier S; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Guadagnin E; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Giangrande PH; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Martini PGV; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA.
  • Vockley J; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, USA. Electronic address: vockleyg@upmc.edu.
Mol Genet Metab ; 138(1): 106982, 2023 01.
Article en En | MEDLINE | ID: mdl-36580829
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Acil-CoA Deshidrogenasa de Cadena Larga / Errores Innatos del Metabolismo Lipídico Límite: Animals / Humans Idioma: En Año: 2023 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Acil-CoA Deshidrogenasa de Cadena Larga / Errores Innatos del Metabolismo Lipídico Límite: Animals / Humans Idioma: En Año: 2023 Tipo del documento: Article