Your browser doesn't support javascript.
loading
Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine model.
Zhao, Xue-Jun; Mohsen, Ai-Walid; Mihalik, Stephanie; Solo, Keaton; Basu, Shakuntala; Aliu, Ermal; Shi, Huifang; Kochersberger, Catherine; Karunanidhi, Anuradha; Van't Land, Clinton; Coughlan, Kimberly A; Siddiqui, Summar; Rice, Lisa M; Hillier, Shawn; Guadagnin, Eleonora; DeAntonis, Christine; Giangrande, Paloma H; Martini, Paolo G V; Vockley, Jerry.
Afiliação
  • Zhao XJ; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Mohsen AW; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Mihalik S; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, 15261, USA.
  • Solo K; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Basu S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Aliu E; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Shi H; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Kochersberger C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Karunanidhi A; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Van't Land C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Coughlan KA; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.
  • Siddiqui S; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Rice LM; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Hillier S; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Guadagnin E; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • DeAntonis C; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Giangrande PH; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Martini PGV; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
  • Vockley J; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA.
Hum Mol Genet ; 32(14): 2347-2356, 2023 07 04.
Article em En | MEDLINE | ID: mdl-37162351
Assuntos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Acil-CoA Desidrogenases / Fibroblastos Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Acil-CoA Desidrogenases / Fibroblastos Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos