Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model-Reference-Cited by-同舟云学术

Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model

Published:2023-01 Issue:1 Volume:138 Page:106982
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

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

Funder

National Institutes of Health

Children's Hospital of Pittsburgh

Moderna

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference49 articles.

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3. Long-chain fatty acid oxidation disorders and current management strategies;Vockley;Am. J. Manag. Care,2020

4. Clinical and biological features at diagnosis in mitochondrial fatty acid beta-oxidation defects: a French pediatric study of 187 patients;Baruteau;J. Inherit. Metab. Dis.,2013

5. Acylcarnitine analysis by tandem mass spectrometry;Smith;Curr. Protoc. Hum. Genet.,2010

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